Somatic Cell Genome Editing (SCGE)
Somatic Cell Genome Editing (SCGE)
Program Publications
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The search results on this publication page are automated on a monthly schedule based on acknowledgement of NIH Common Fund award numbers and intramural awards. Therefore, this list is not an exhaustive or error-free account of the program’s publications.
| Title | Author | Journal Name | PubMedID | Journal Abbreviation | Publication Date |
|---|---|---|---|---|---|
| Nanoparticle single-cell multiomic readouts reveal that cell heterogeneity influences lipid nanoparticle-mediated messenger RNA delivery. | Dobrowolski C, Paunovska K, Schrader Echeverri E, Loughrey D, Da Silva Sanchez AJ, Ni H, Hatit MZC, Lokugamage MP, Kuzminich Y, Peck HE, Santangelo PJ, Dahlman JE | Nature nanotechnology | 35768613 | Nat Nanotechnol | 2022 Aug |
| Nanoparticles containing constrained phospholipids deliver mRNA to liver immune cells in vivo without targeting ligands. | Gan Z, Lokugamage MP, Hatit MZC, Loughrey D, Paunovska K, Sato M, Cristian A, Dahlman JE | Bioengineering & translational medicine | 33758781 | Bioeng Transl Med | 2020 Sep |
| Therapeutic RNA Delivery for COVID and Other Diseases. | Dobrowolski C, Paunovska K, Hatit MZC, Lokugamage MP, Dahlman JE | Advanced healthcare materials | 33661555 | Adv Healthc Mater | 2021 Aug |
| Lipid nanoparticle-mediated mRNA delivery to CD34(+) cells in rhesus monkeys. | Kim H, Zenhausern R, Gentry K, Lian L, Huayamares SG, Radmand A, Loughrey D, Podilapu AR, Hatit MZC, Ni H, Li A, Shajii A, Peck HE, Han K, Hua X, Jia S, Martinez M, Lee C, Santangelo PJ, Tarantal A, Dahlman JE | Nature biotechnology | 39578569 | Nat Biotechnol | 2024 Nov 22 |
| Substituting racemic ionizable lipids with stereopure ionizable lipids can increase mRNA delivery. | Da Silva Sanchez AJ, Zhao K, Huayamares SG, Hatit MZC, Lokugamage MP, Loughrey D, Dobrowolski C, Wang S, Kim H, Paunovska K, Kuzminich Y, Dahlman JE | Journal of controlled release : official journal of the Controlled Release Society | 36423872 | J Control Release | 2023 Jan |
| Cationic cholesterol-dependent LNP delivery to lung stem cells, the liver, and heart. | Radmand A, Kim H, Beyersdorf J, Dobrowolski CN, Zenhausern R, Paunovska K, Huayamares SG, Hua X, Han K, Loughrey D, Hatit MZC, Del Cid A, Ni H, Shajii A, Li A, Muralidharan A, Peck HE, Tiegreen KE, Jia S, Santangelo PJ, Dahlman JE | Proceedings of the National Academy of Sciences of the United States of America | 38437539 | Proc Natl Acad Sci U S A | 2024 Mar 12 |
| Species-dependent in vivo mRNA delivery and cellular responses to nanoparticles. | Hatit MZC, Lokugamage MP, Dobrowolski CN, Paunovska K, Ni H, Zhao K, Vanover D, Beyersdorf J, Peck HE, Loughrey D, Sato M, Cristian A, Santangelo PJ, Dahlman JE | Nature nanotechnology | 35132167 | Nat Nanotechnol | 2022 Mar |
| Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungs. | Lokugamage MP, Vanover D, Beyersdorf J, Hatit MZC, Rotolo L, Echeverri ES, Peck HE, Ni H, Yoon JK, Kim Y, Santangelo PJ, Dahlman JE | Nature biomedical engineering | 34616046 | Nat Biomed Eng | 2021 Sep |
| Nanoparticle stereochemistry-dependent endocytic processing improves in vivo mRNA delivery. | Hatit MZC, Dobrowolski CN, Lokugamage MP, Loughrey D, Ni H, Zurla C, Da Silva Sanchez AJ, Radmand A, Huayamares SG, Zenhausern R, Paunovska K, Peck HE, Kim J, Sato M, Feldman JI, Rivera MA, Cristian A, Kim Y, Santangelo PJ, Dahlman JE | Nature chemistry | 36864143 | Nat Chem | 2023 Apr |
| A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver. | Zheng C, Liang SQ, Liu B, Liu P, Kwan SY, Wolfe SA, Xue W | Molecular therapy : the journal of the American Society of Gene Therapy | 34998953 | Mol Ther | 2022 Mar 2 |
| Reducing the inherent auto-inhibitory interaction within the pegRNA enhances prime editing efficiency. | Ponnienselvan K, Liu P, Nyalile T, Oikemus S, Maitland SA, Lawson ND, Luban J, Wolfe SA | Nucleic acids research | 37246708 | Nucleic Acids Res | 2023 Jul 21 |
| Template-jumping prime editing enables large insertion and exon rewriting in vivo. | Zheng C, Liu B, Dong X, Gaston N, Sontheimer EJ, Xue W | Nature communications | 37291100 | Nat Commun | 2023 Jun 8 |
| Genome-wide profiling of prime editor off-target sites in vitro and in vivo using PE-tag. | Liang SQ, Liu P, Ponnienselvan K, Suresh S, Chen Z, Kramme C, Chatterjee P, Zhu LJ, Sontheimer EJ, Xue W, Wolfe SA | Nature methods | 37156841 | Nat Methods | 2023 Jun |
| Nucleic Acid Therapeutics for Neurological Diseases. | Watts JK, Brown RH, Khvorova A | Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics | 31037650 | Neurotherapeutics | 2019 Apr |
| Efficient Homology-Directed Repair with Circular Single-Stranded DNA Donors. | Iyer S, Mir A, Vega-Badillo J, Roscoe BP, Ibraheim R, Zhu LJ, Lee J, Liu P, Luk K, Mintzer E, Guo D, Soares de Brito J, Emerson CP Jr, Zamore PD, Sontheimer EJ, Wolfe SA | The CRISPR journal | 36070530 | CRISPR J | 2022 Oct |
| Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo. | Ibraheim R, Tai PWL, Mir A, Javeed N, Wang J, Rodríguez TC, Namkung S, Nelson S, Khokhar ES, Mintzer E, Maitland S, Chen Z, Cao Y, Tsagkaraki E, Wolfe SA, Wang D, Pai AA, Xue W, Gao G, Sontheimer EJ | Nature communications | 34725353 | Nat Commun | 2021 Nov 1 |
| The NIH Somatic Cell Genome Editing program. | Saha K, Sontheimer EJ, Brooks PJ, Dwinell MR, Gersbach CA, Liu DR, Murray SA, Tsai SQ, Wilson RC, Anderson DG, Asokan A, Banfield JF, Bankiewicz KS, Bao G, Bulte JWM, Bursac N, Campbell JM, Carlson DF, Chaikof EL, Chen ZY, Cheng RH, Clark KJ, Curiel DT, Dahlman JE, Deverman BE, Dickinson ME, Doudna JA, Ekker SC, Emborg ME, Feng G, Freedman BS, Gamm DM, Gao G, Ghiran IC, Glazer PM, Gong S, Heaney JD, Hennebold JD, Hinson JT, Khvorova A, Kiani S, Lagor WR, Lam KS, Leong KW, Levine JE, Lewis JA, Lutz CM, Ly DH, Maragh S, McCray PB Jr, McDevitt TC, Mirochnitchenko O, Morizane R, Murthy N, Prather RS, Ronald JA, Roy S, Roy S, Sabbisetti V, Saltzman WM, Santangelo PJ, Segal DJ, Shimoyama M, Skala MC, Tarantal AF, Tilton JC, Truskey GA, Vandsburger M, Watts JK, Wells KD, Wolfe SA, Xu Q, Xue W, Yi G, Zhou J, SCGE Consortium | Nature | 33828315 | Nature | 2021 Apr |
| Increasing intracellular dNTP levels improves prime editing efficiency. | Liu P, Ponnienselvan K, Nyalile T, Oikemus S, Joynt AT, Iyer S, Kelly K, Guo D, Kyawe PP, Vanderleeden E, Redick SD, Huang L, Chen Z, Lee JM, Schiffer CA, Harlan DM, Wang JP, Emerson CP Jr, Lawson ND, Watts JK, Sontheimer EJ, Luban J, Wolfe SA | Nature biotechnology | 39322763 | Nat Biotechnol | 2024 Sep 25 |
| Double Click: Unexpected 1:2 Stoichiometry in a Norbornene-Tetrazine Reaction. | Devi G, Hedger AK, Whitby RJ, Watts JK | The Journal of organic chemistry | 37058436 | J Org Chem | 2023 May 5 |
| Capturing prime editor off-target sites within the genome. | Nature methods | 37156844 | Nat Methods | 2023 Jun | |
| Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice. | Liu P, Liang SQ, Zheng C, Mintzer E, Zhao YG, Ponnienselvan K, Mir A, Sontheimer EJ, Gao G, Flotte TR, Wolfe SA, Xue W | Nature communications | 33837189 | Nat Commun | 2021 Apr 9 |
| Genome-wide detection of CRISPR editing in vivo using GUIDE-tag. | Liang SQ, Liu P, Smith JL, Mintzer E, Maitland S, Dong X, Yang Q, Lee J, Haynes CM, Zhu LJ, Watts JK, Sontheimer EJ, Wolfe SA, Xue W | Nature communications | 35064134 | Nat Commun | 2022 Jan 21 |
| Tetrazine-Ligated CRISPR sgRNAs for Efficient Genome Editing. | Chen Z, Devi G, Arif A, Zamore PD, Sontheimer EJ, Watts JK | ACS chemical biology | 35446558 | ACS Chem Biol | 2022 May 20 |
| A Fluorescent Reporter Mouse for In Vivo Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editors. | Chen Z, Kwan SY, Mir A, Hazeltine M, Shin M, Liang SQ, Chan IL, Kelly K, Ghanta KS, Gaston N, Cao Y, Xie J, Gao G, Xue W, Sontheimer EJ, Watts JK | The CRISPR journal | 38108517 | CRISPR J | 2023 Dec |
| In Vivo Prime Editing by Lipid Nanoparticle Co-delivery of Chemically Modified pegRNA and Prime Editor mRNA. | Chen Z, Kelly K, Cheng H, Dong X, Hedger AK, Li L, Sontheimer EJ, Watts JK | GEN biotechnology | 39850578 | GEN Biotechnol | 2023 Dec |
| Structures of Neisseria meningitidis Cas9 Complexes in Catalytically Poised and Anti-CRISPR-Inhibited States. | Sun W, Yang J, Cheng Z, Amrani N, Liu C, Wang K, Ibraheim R, Edraki A, Huang X, Wang M, Wang J, Liu L, Sheng G, Yang Y, Lou J, Sontheimer EJ, Wang Y | Molecular cell | 31668930 | Mol Cell | 2019 Dec 19 |
| Enhanced Cas12a editing in mammalian cells and zebrafish. | Liu P, Luk K, Shin M, Idrizi F, Kwok S, Roscoe B, Mintzer E, Suresh S, Morrison K, Frazão JB, Bolukbasi MF, Ponnienselvan K, Luban J, Zhu LJ, Lawson ND, Wolfe SA | Nucleic acids research | 30892626 | Nucleic Acids Res | 2019 May 7 |
| 5'-Modifications improve potency and efficacy of DNA donors for precision genome editing. | Ghanta KS, Chen Z, Mir A, Dokshin GA, Krishnamurthy PM, Yoon Y, Gallant J, Xu P, Zhang XO, Ozturk AR, Shin M, Idrizi F, Liu P, Gneid H, Edraki A, Lawson ND, Rivera-Pérez JA, Sontheimer EJ, Watts JK, Mello CC | eLife | 34665130 | Elife | 2021 Oct 19 |
| CRISPR-enhanced human adipocyte browning as cell therapy for metabolic disease. | Tsagkaraki E, Nicoloro SM, DeSouza T, Solivan-Rivera J, Desai A, Lifshitz LM, Shen Y, Kelly M, Guilherme A, Henriques F, Amrani N, Ibraheim R, Rodriguez TC, Luk K, Maitland S, Friedline RH, Tauer L, Hu X, Kim JK, Wolfe SA, Sontheimer EJ, Corvera S, Czech MP | Nature communications | 34836963 | Nat Commun | 2021 Nov 26 |
| Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells. | Nagpal N, Wang J, Zeng J, Lo E, Moon DH, Luk K, Braun RO, Burroughs LM, Keel SB, Reilly C, Lindsley RC, Wolfe SA, Tai AK, Cahan P, Bauer DE, Fong YW, Agarwal S | Cell stem cell | 32320679 | Cell Stem Cell | 2020 Jun 4 |
| Crosstalk between corepressor NRIP1 and cAMP signaling on adipocyte thermogenic programming. | Tsagkaraki E, Guilherme A, Nicoloro SM, Kelly M, Lifshitz LM, Wang H, Min K, Rowland LA, Santos KB, Wetoska N, Friedline RH, Maitland SA, Chen M, Weinstein LS, Wolfe SA, Kim JK, Czech MP | Molecular metabolism | 37482187 | Mol Metab | 2023 Oct |
| Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo. | Zhang H, Kelly K, Lee J, Echeverria D, Cooper D, Panwala R, Amrani N, Chen Z, Gaston N, Wagh A, Newby GA, Xie J, Liu DR, Gao G, Wolfe SA, Khvorova A, Watts JK, Sontheimer EJ | Nucleic acids research | 38033325 | Nucleic Acids Res | 2024 Jan 25 |
| Optimization of Nuclear Localization Signal Composition Improves CRISPR-Cas12a Editing Rates in Human Primary Cells. | Luk K, Liu P, Zeng J, Wang Y, Maitland SA, Idrizi F, Ponnienselvan K, Iyer S, Zhu LJ, Luban J, Bauer DE, Wolfe SA | GEN biotechnology | 38405215 | GEN Biotechnol | 2022 Jun |
| Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo. | Zhang H, Kelly K, Lee J, Echeverria D, Cooper D, Panwala R, Chen Z, Gaston N, Newby GA, Xie J, Liu DR, Gao G, Wolfe SA, Khvorova A, Watts JK, Sontheimer EJ | bioRxiv : the preprint server for biology | 36993169 | bioRxiv | 2023 Mar 23 |
| A brown fat-enriched adipokine, ASRA, is a leptin receptor antagonist that stimulates appetite. | Huang L, Liu P, Du Y, Bazan JF, Pan D, Chen Q, Lee A, Kola VSR, Wolfe SA, Wang YX | bioRxiv : the preprint server for biology | 37745491 | bioRxiv | 2024 Nov 17 |
| Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosis. | Qiu M, Tang Y, Chen J, Muriph R, Ye Z, Huang C, Evans J, Henske EP, Xu Q | Proceedings of the National Academy of Sciences of the United States of America | 35173043 | Proc Natl Acad Sci U S A | 2022 Feb 22 |
| Intracellular Delivery of His-Tagged Genome-Editing Proteins Enabled by Nitrilotriacetic Acid-Containing Lipidoid Nanoparticles. | Li Y, Li AC, Xu Q | Advanced healthcare materials | 30565897 | Adv Healthc Mater | 2019 Mar |
| Developing Biodegradable Lipid Nanoparticles for Intracellular mRNA Delivery and Genome Editing. | Qiu M, Li Y, Bloomer H, Xu Q | Accounts of chemical research | 34668716 | Acc Chem Res | 2021 Nov 2 |
| Harnessing non-Watson-Crick's base pairing to enhance CRISPR effectors cleavage activities and enable gene editing in mammalian cells. | Gao S, Guan H, Bloomer H, Wich D, Song D, Khirallah J, Ye Z, Zhao Y, Chen M, Xu C, Liu L, Xu Q | Proceedings of the National Academy of Sciences of the United States of America | 38150477 | Proc Natl Acad Sci U S A | 2024 Jan 9 |
| Tuning Lipid Nanoparticles for RNA Delivery to Extrahepatic Organs. | Song D, Zhao Y, Wang Z, Xu Q | Advanced materials (Deerfield Beach, Fla.) | 39233550 | Adv Mater | 2024 Nov |
| Targeted genome editing restores auditory function in adult mice with progressive hearing loss caused by a human microRNA mutation. | Zhu W, Du W, Rameshbabu AP, Armstrong AM, Silver S, Kim Y, Wei W, Shu Y, Liu X, Lewis MA, Steel KP, Chen ZY | Science translational medicine | 38985856 | Sci Transl Med | 2024 Jul 10 |
| Nonviral Nanoparticles for CRISPR-Based Genome Editing: Is It Just a Simple Adaption of What Have Been Developed for Nucleic Acid Delivery? | Qiu M, Glass Z, Xu Q | Biomacromolecules | 31342740 | Biomacromolecules | 2019 Sep 9 |
| Protein and mRNA Delivery Enabled by Cholesteryl-Based Biodegradable Lipidoid Nanoparticles. | Li Y, Jarvis R, Zhu K, Glass Z, Ogurlu R, Gao P, Li P, Chen J, Yu Y, Yang Y, Xu Q | Angewandte Chemie (International ed. in English) | 32438474 | Angew Chem Int Ed Engl | 2020 Aug 24 |
| Imidazole-Based Synthetic Lipidoids for In Vivo mRNA Delivery into Primary T Lymphocytes. | Zhao X, Chen J, Qiu M, Li Y, Glass Z, Xu Q | Angewandte Chemie (International ed. in English) | 32662132 | Angew Chem Int Ed Engl | 2020 Nov 2 |
| Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors. | Krishnamurthy S, Traore S, Cooney AL, Brommel CM, Kulhankova K, Sinn PL, Newby GA, Liu DR, McCray PB | Nucleic acids research | 34520545 | Nucleic Acids Res | 2021 Oct 11 |
| Reprogramming by drug-like molecules leads to regeneration of cochlear hair cell-like cells in adult mice. | Quan YZ, Wei W, Ergin V, Rameshbabu AP, Huang M, Tian C, Saladi SV, Indzhykulian AA, Chen ZY | Proceedings of the National Academy of Sciences of the United States of America | 37068229 | Proc Natl Acad Sci U S A | 2023 Apr 25 |
| Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8. | Du W, Ergin V, Loeb C, Huang M, Silver S, Armstrong AM, Huang Z, Gurumurthy CB, Staecker H, Liu X, Chen ZY | Molecular therapy : the journal of the American Society of Gene Therapy | 37244253 | Mol Ther | 2023 Sep 6 |
| In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness. | Yeh WH, Shubina-Oleinik O, Levy JM, Pan B, Newby GA, Wornow M, Burt R, Chen JC, Holt JR, Liu DR | Science translational medicine | 32493795 | Sci Transl Med | 2020 Jun 3 |
| Combinatorial Library of Cyclic Benzylidene Acetal-Containing pH-Responsive Lipidoid Nanoparticles for Intracellular mRNA Delivery. | Li Y, Li R, Chakraborty A, Ogurlu R, Zhao X, Chen J, Xu Q | Bioconjugate chemistry | 32520527 | Bioconjug Chem | 2020 Jul 15 |
| CRISPR/Cas9 ribonucleoprotein-mediated genome and epigenome editing in mammalian cells. | Bloomer H, Khirallah J, Li Y, Xu Q | Advanced drug delivery reviews | 34942274 | Adv Drug Deliv Rev | 2022 Feb |
| Gene editing in a Myo6 semi-dominant mouse model rescues auditory function. | Xue Y, Hu X, Wang D, Li D, Li Y, Wang F, Huang M, Gu X, Xu Z, Zhou J, Wang J, Chai R, Shen J, Chen ZY, Li GL, Yang H, Li H, Zuo E, Shu Y | Molecular therapy : the journal of the American Society of Gene Therapy | 34174443 | Mol Ther | 2022 Jan 5 |
| Integrating Combinatorial Lipid Nanoparticle and Chemically Modified Protein for Intracellular Delivery and Genome Editing. | Chang J, Chen X, Glass Z, Gao F, Mao L, Wang M, Xu Q | Accounts of chemical research | 30586281 | Acc Chem Res | 2019 Mar 19 |
| Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles. | Liu J, Chang J, Jiang Y, Meng X, Sun T, Mao L, Xu Q, Wang M | Advanced materials (Deerfield Beach, Fla.) | 31215123 | Adv Mater | 2019 Aug |
| mRNA Delivery Using Bioreducible Lipidoid Nanoparticles Facilitates Neural Differentiation of Human Mesenchymal Stem Cells. | Zhao X, Glass Z, Chen J, Yang L, Kaplan DL, Xu Q | Advanced healthcare materials | 32815325 | Adv Healthc Mater | 2021 Feb |
| Improving adenine base editing precision by enlarging the recognition domain of CRISPR-Cas9. | Gao S, Weng B, Wich D, Power L, Chen M, Guan H, Ye Z, Xu C, Xu Q | Nature communications | 40021632 | Nat Commun | 2025 Feb 28 |
| Generation and Genetic Correction of USH2A c.2299delG Mutation in Patient-Derived Induced Pluripotent Stem Cells. | Liu X, Lillywhite J, Zhu W, Huang Z, Clark AM, Gosstola N, Maguire CT, Dykxhoorn D, Chen ZY, Yang J | Genes | 34070435 | Genes (Basel) | 2021 May 25 |
| Direct Functional Protein Delivery with a Peptide into Neonatal and Adult Mammalian Inner Ear In Vivo. | Zhang K, Cheng X, Zhao L, Huang M, Tao Y, Zhang H, Rosenholm JM, Zhuang M, Chen ZY, Chen B, Shu Y | Molecular therapy. Methods & clinical development | 32953927 | Mol Ther Methods Clin Dev | 2020 Sep 11 |
| Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3. | Qiu M, Glass Z, Chen J, Haas M, Jin X, Zhao X, Rui X, Ye Z, Li Y, Zhang F, Xu Q | Proceedings of the National Academy of Sciences of the United States of America | 33649229 | Proc Natl Acad Sci U S A | 2021 Mar 9 |
| In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice. | Koblan LW, Erdos MR, Wilson C, Cabral WA, Levy JM, Xiong ZM, Tavarez UL, Davison LM, Gete YG, Mao X, Newby GA, Doherty SP, Narisu N, Sheng Q, Krilow C, Lin CY, Gordon LB, Cao K, Collins FS, Brown JD, Liu DR | Nature | 33408413 | Nature | 2021 Jan |
| Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression. | Gao P, Lyu Q, Ghanam AR, Lazzarotto CR, Newby GA, Zhang W, Choi M, Slivano OJ, Holden K, Walker JA 2nd, Kadina AP, Munroe RJ, Abratte CM, Schimenti JC, Liu DR, Tsai SQ, Long X, Miano JM | Genome biology | 33722289 | Genome Biol | 2021 Mar 16 |
| Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses. | Levy JM, Yeh WH, Pendse N, Davis JR, Hennessey E, Butcher R, Koblan LW, Comander J, Liu Q, Liu DR | Nature biomedical engineering | 31937940 | Nat Biomed Eng | 2020 Jan |
| Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications. | Li Y, Glass Z, Huang M, Chen ZY, Xu Q | Biomaterials | 31945616 | Biomaterials | 2020 Mar |
| Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection. | Ma F, Yang L, Sun Z, Chen J, Rui X, Glass Z, Xu Q | Science advances | 32832671 | Sci Adv | 2020 Jul |
| Clinical progress in genome-editing technology and in vivo delivery techniques. | Khirallah J, Eimbinder M, Li Y, Xu Q | Trends in genetics : TIG | 36669950 | Trends Genet | 2023 Mar |
| Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo. | Tao Y, Lamas V, Du W, Zhu W, Li Y, Whittaker MN, Zuris JA, Thompson DB, Rameshbabu AP, Shu Y, Gao X, Hu JH, Pei C, Kong WJ, Liu X, Wu H, Kleinstiver BP, Liu DR, Chen ZY | Nature communications | 37582836 | Nat Commun | 2023 Aug 15 |
| Gene editing therapeutics based on mRNA delivery. | Popovitz J, Sharma R, Hoshyar R, Soo Kim B, Murthy N, Lee K | Advanced drug delivery reviews | 37516409 | Adv Drug Deliv Rev | 2023 Sep |
| Advances in acid-degradable and enzyme-cleavable linkers for drug delivery. | Zhao S, Yu N, Han H, Guo S, Murthy N | Current opinion in chemical biology | 39642424 | Curr Opin Chem Biol | 2025 Feb |
| A self-immolative linker that releases thiols detects penicillin amidase and nitroreductase with high sensitivity via absorption spectroscopy. | Espinoza EM, Røise JJ, He M, Li IC, Agatep AK, Udenyi P, Han H, Jackson N, Kerr DL, Chen D, Stentzel MR, Ruan E, Riley L, Murthy N | Chemical communications (Cambridge, England) | 35170593 | Chem Commun (Camb) | 2022 Mar 3 |
| Mercapto-pyrimidines are reversible covalent inhibitors of the papain-like protease (PLpro) and inhibit SARS-CoV-2 (SCoV-2) replication. | Bajaj T, Wehri E, Suryawanshi RK, King E, Pardeshi KS, Behrouzi K, Khodabakhshi Z, Schulze-Gahmen U, Kumar GR, Mofrad MRK, Nomura DK, Ott M, Schaletzky J, Murthy N | RSC advances | 37312993 | RSC Adv | 2023 Jun 9 |
| Acid-Sensitive Surfactants Enhance the Delivery of Nucleic Acids. | Røise JJ, Han H, Li J, Kerr DL, Taing C, Behrouzi K, He M, Ruan E, Chan LY, Espinoza EM, Reinhard S, Thakker K, Kwon J, Mofrad MRK, Murthy N | Molecular pharmaceutics | 34931518 | Mol Pharm | 2022 Jan 3 |
| Lipid Nanoparticles Deliver mRNA to the Brain after an Intracerebral Injection. | Tuma J, Chen YJ, Collins MG, Paul A, Li J, Han H, Sharma R, Murthy N, Lee HY | Biochemistry | 37729550 | Biochemistry | 2023 Dec 19 |
| The Coiled-Coil Forming Peptide (KVSALKE)(5) Is a Cell Penetrating Peptide that Enhances the Intracellular Delivery of Proteins. | Li J, Tuma J, Han H, Kim H, Wilson RC, Lee HY, Murthy N | Advanced healthcare materials | 34861744 | Adv Healthc Mater | 2022 May |
| The delivery challenge: fulfilling the promise of therapeutic genome editing. | van Haasteren J, Li J, Scheideler OJ, Murthy N, Schaffer DV | Nature biotechnology | 32601435 | Nat Biotechnol | 2020 Jul |
| In utero delivery of mRNA to the heart, diaphragm and muscle with lipid nanoparticles. | Gao K, Li J, Song H, Han H, Wang Y, Yin B, Farmer DL, Murthy N, Wang A | Bioactive materials | 36844366 | Bioact Mater | 2023 Jul |
| Design of therapeutic biomaterials to control inflammation. | Tu Z, Zhong Y, Hu H, Shao D, Haag R, Schirner M, Lee J, Sullenger B, Leong KW | Nature reviews. Materials | 35251702 | Nat Rev Mater | 2022 |
| Material Engineering in Gut Microbiome and Human Health. | Yang L, Hung LY, Zhu Y, Ding S, Margolis KG, Leong KW | Research (Washington, D.C.) | 35958108 | Research (Wash D C) | 2022 |
| Advances in microphysiological blood-brain barrier (BBB) models towards drug delivery. | Lee CS, Leong KW | Current opinion in biotechnology | 32711361 | Curr Opin Biotechnol | 2020 Dec |
| A Versatile and Robust Platform for the Scalable Manufacture of Biomimetic Nanovaccines. | Hu H, Yang C, Zhang F, Li M, Tu Z, Mu L, Dawulieti J, Lao YH, Xiao Z, Yan H, Sun W, Shao D, Leong KW | Advanced science (Weinheim, Baden-Wurttemberg, Germany) | 34386315 | Adv Sci (Weinh) | 2021 Aug |
| Light: A Magical Tool for Controlled Drug Delivery. | Tao Y, Chan HF, Shi B, Li M, Leong KW | Advanced functional materials | 34483808 | Adv Funct Mater | 2020 Dec 1 |
| Phenylboronic Acid-Functionalized Polyplexes Tailored to Oral CRISPR Delivery. | Yoshinaga N, Zhou JK, Xu C, Quek CH, Zhu Y, Tang D, Hung LY, Najjar SA, Shiu CYA, Margolis KG, Lao YH, Leong KW | Nano letters | 36648291 | Nano Lett | 2023 Feb 8 |
| Scalable projected Light Sheet Microscopy for high-resolution imaging of living and cleared samples. | Chen Y, Chauhan S, Gong C, Dayton H, Xu C, De La Cruz ED, Datta MS, Leong KW, Dietrich LEP, Tomer R | bioRxiv : the preprint server for biology | 37333196 | bioRxiv | 2023 Jul 18 |
| Multiscale engineering of brain organoids for disease modeling. | Xu C, Alameri A, Leong W, Johnson E, Chen Z, Xu B, Leong KW | Advanced drug delivery reviews | 38810702 | Adv Drug Deliv Rev | 2024 Jul |
| Focused ultrasound-mediated brain genome editing. | Lao YH, Ji R, Zhou JK, Snow KJ, Kwon N, Saville E, He S, Chauhan S, Chi CW, Datta MS, Zhang H, Quek CH, Cai SS, Li M, Gaitan Y, Bechtel L, Wu SY, Lutz CM, Tomer R, Murray SA, Chavez A, Konofagou EE, Leong KW | Proceedings of the National Academy of Sciences of the United States of America | 37579143 | Proc Natl Acad Sci U S A | 2023 Aug 22 |
| Focused ultrasound-mediated brain genome editing. | Leong K, Lao YH, Ji R, Zhou J, Snow K, Kwon N, Saville E, He S, Chauhan S, Chi CW, Datta M, Zhang H, Quek CH, Cai S, Li M, Gaitan Y, Bechtel L, Wu SY, Lutz C, Tomer R, Murray S, Chavez A, Konofagou E | Research square | 36712096 | Res Sq | 2023 Jan 20 |
| Cortical overgrowth in a preclinical forebrain organoid model of CNTNAP2-associated autism spectrum disorder. | de Jong JO, Llapashtica C, Genestine M, Strauss K, Provenzano F, Sun Y, Zhu H, Cortese GP, Brundu F, Brigatti KW, Corneo B, Migliori B, Tomer R, Kushner SA, Kellendonk C, Javitch JA, Xu B, Markx S | Nature communications | 34471112 | Nat Commun | 2021 Sep 1 |
| Flash Technology-Based Self-Assembly in Nanoformulation: From Fabrication to Biomedical Applications. | Hu H, Yang C, Li M, Shao D, Mao HQ, Leong KW | Materials today (Kidlington, England) | 34421329 | Mater Today (Kidlington) | 2021 Jan-Feb |
| A Versatile Nonviral Delivery System for Multiplex Gene-Editing in the Liver. | Gong J, Wang HX, Lao YH, Hu H, Vatan N, Guo J, Ho TC, Huang D, Li M, Shao D, Leong KW | Advanced materials (Deerfield Beach, Fla.) | 33053221 | Adv Mater | 2020 Nov |
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| 3D cell culture models: Drug pharmacokinetics, safety assessment, and regulatory consideration. | Wang H, Brown PC, Chow ECY, Ewart L, Ferguson SS, Fitzpatrick S, Freedman BS, Guo GL, Hedrich W, Heyward S, Hickman J, Isoherranen N, Li AP, Liu Q, Mumenthaler SM, Polli J, Proctor WR, Ribeiro A, Wang JY, Wange RL, Huang SM | Clinical and translational science | 33982436 | Clin Transl Sci | 2021 Sep |
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| Serum Concentrations of Per- and Polyfluoroalkyl Substances and Risk of Renal Cell Carcinoma. | Shearer JJ, Callahan CL, Calafat AM, Huang WY, Jones RR, Sabbisetti VS, Freedman ND, Sampson JN, Silverman DT, Purdue MP, Hofmann JN | Journal of the National Cancer Institute | 32944748 | J Natl Cancer Inst | 2021 May 4 |
| A parathyroid hormone/salt-inducible kinase signaling axis controls renal vitamin D activation and organismal calcium homeostasis. | Yoon SH, Meyer MB, Arevalo C, Tekguc M, Zhang C, Wang JS, Castro Andrade CD, Strauss K, Sato T, Benkusky NA, Lee SM, Berdeaux R, Foretz M, Sundberg TB, Xavier RJ, Adelmann CH, Brooks DJ, Anselmo A, Sadreyev RI, Rosales IA, Fisher DE, Gupta N, Morizane R, Greka A, Pike JW, Mannstadt M, Wein MN | The Journal of clinical investigation | 36862513 | J Clin Invest | 2023 May 1 |
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| Genetics of cystogenesis in base-edited human organoids reveal therapeutic strategies for polycystic kidney disease. | Vishy CE, Thomas C, Vincent T, Crawford DK, Goddeeris MM, Freedman BS | Cell stem cell | 38579684 | Cell Stem Cell | 2024 Apr 4 |
| Interferon-γ induces combined pyroptotic angiopathy and APOL1 expression in human kidney disease. | Juliar BA, Stanaway IB, Sano F, Fu H, Smith KD, Akilesh S, Scales SJ, El Saghir J, Bhatraju PK, Liu E, Yang J, Lin J, Eddy S, Kretzler M, Zheng Y, Himmelfarb J, Harder JL, Freedman BS | Cell reports | 38838223 | Cell Rep | 2024 Jun 25 |
| Adult human kidney organoids originate from CD24(+) cells and represent an advanced model for adult polycystic kidney disease. | Xu Y, Kuppe C, Perales-Patón J, Hayat S, Kranz J, Abdallah AT, Nagai J, Li Z, Peisker F, Saritas T, Halder M, Menzel S, Hoeft K, Kenter A, Kim H, van Roeyen CRC, Lehrke M, Moellmann J, Speer T, Buhl EM, Hoogenboezem R, Boor P, Jansen J, Knopp C, Kurth I, Smeets B, Bindels E, Reinders MEJ, Baan C, Gribnau J, Hoorn EJ, Steffens J, Huber TB, Costa I, Floege J, Schneider RK, Saez-Rodriguez J, Freedman BS, Kramann R | Nature genetics | 36303074 | Nat Genet | 2022 Nov |
| Multivalent designed proteins neutralize SARS-CoV-2 variants of concern and confer protection against infection in mice. | Hunt AC, Case JB, Park YJ, Cao L, Wu K, Walls AC, Liu Z, Bowen JE, Yeh HW, Saini S, Helms L, Zhao YT, Hsiang TY, Starr TN, Goreshnik I, Kozodoy L, Carter L, Ravichandran R, Green LB, Matochko WL, Thomson CA, Vögeli B, Krüger A, VanBlargan LA, Chen RE, Ying B, Bailey AL, Kafai NM, Boyken SE, Ljubetič A, Edman N, Ueda G, Chow CM, Johnson M, Addetia A, Navarro MJ, Panpradist N, Gale M Jr, Freedman BS, Bloom JD, Ruohola-Baker H, Whelan SPJ, Stewart L, Diamond MS, Veesler D, Jewett MC, Baker D | Science translational medicine | 35412328 | Sci Transl Med | 2022 May 25 |
| Recommendations on fit-for-purpose criteria to establish quality management for microphysiological systems and for monitoring their reproducibility. | Pamies D, Ekert J, Zurich MG, Frey O, Werner S, Piergiovanni M, Freedman BS, Keong Teo AK, Erfurth H, Reyes DR, Loskill P, Candarlioglu P, Suter-Dick L, Wang S, Hartung T, Coecke S, Stacey GN, Wagegg BA, Dehne EM, Pistollato F, Leist M | Stem cell reports | 38959889 | Stem Cell Reports | 2024 Jul 2 |
| Cross-validation of SARS-CoV-2 responses in kidney organoids and clinical populations. | Helms L, Marchiano S, Stanaway IB, Hsiang TY, Juliar BA, Saini S, Zhao YT, Khanna A, Menon R, Alakwaa F, Mikacenic C, Morrell ED, Wurfel MM, Kretzler M, Harder JL, Murry CE, Himmelfarb J, Ruohola-Baker H, Bhatraju PK, Gale M Jr, Freedman BS | JCI insight | 34767537 | JCI Insight | 2021 Dec 22 |
| Glucose absorption drives cystogenesis in a human organoid-on-chip model of polycystic kidney disease. | Li SR, Gulieva RE, Helms L, Cruz NM, Vincent T, Fu H, Himmelfarb J, Freedman BS | Nature communications | 36564419 | Nat Commun | 2022 Dec 23 |
| Cyclin G1 induces maladaptive proximal tubule cell dedifferentiation and renal fibrosis through CDK5 activation. | Taguchi K, Elias BC, Sugahara S, Sant S, Freedman BS, Waikar SS, Pozzi A, Zent R, Harris RC, Parikh SM, Brooks CR | The Journal of clinical investigation | 36453545 | J Clin Invest | 2022 Dec 1 |
| Recommendations on fit-for-purpose criteria to establish quality management for microphysiological systems and for monitoring their reproducibility. | Pamies D, Ekert J, Zurich MG, Frey O, Werner S, Piergiovanni M, Freedman BS, Keong Teo AK, Erfurth H, Reyes DR, Loskill P, Candarlioglu P, Suter-Dick L, Wang S, Hartung T, Coecke S, Stacey GN, Wagegg BA, Dehne EM, Pistollato F, Leist M | Stem cell reports | 38670111 | Stem Cell Reports | 2024 May 14 |
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| CHANGE-seq-BE enables simultaneously sensitive and unbiased in vitro profiling of base editor genome-wide activity. | Lazzarotto CR, Katta V, Li Y, Urbina E, Lee G, Tsai SQ | bioRxiv : the preprint server for biology | 38585919 | bioRxiv | 2024 Mar 30 |
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| Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy. | Alves CRR, Ha LL, Yaworski R, Sutton ER, Lazzarotto CR, Christie KA, Reilly A, Beauvais A, Doll RM, de la Cruz D, Maguire CA, Swoboda KJ, Tsai SQ, Kothary R, Kleinstiver BP | Nature biomedical engineering | 38057426 | Nat Biomed Eng | 2024 Feb |
| Virus-free CRISPR knock-in of a chimeric antigen receptor into KLRC1 generates potent GD2-specific natural killer cells. | Shankar K, Zingler-Hoslet I, Shi L, Katta V, Russell BE, Tsai SQ, Capitini CM, Saha K | bioRxiv : the preprint server for biology | 38405747 | bioRxiv | 2024 Feb 14 |
| Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34(+) cell therapy to induce fetal hemoglobin for sickle cell disease. | Katta V, O'Keefe K, Li Y, Mayuranathan T, Lazzarotto CR, Wood RK, Levine RM, Powers A, Mayberry K, Manquen G, Yao Y, Zhang J, Jang Y, Nimmagadda N, Dempsey EA, Lee G, Uchida N, Cheng Y, Fazio F, Lockey T, Meagher M, Sharma A, Tisdale JF, Zhou S, Yen JS, Weiss MJ, Tsai SQ | Molecular therapy : the journal of the American Society of Gene Therapy | 39086133 | Mol Ther | 2024 Oct 2 |
| High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease. | Bzhilyanskaya V, Ma L, Liu S, Fox LR, Whittaker MN, Meis RJ, Choi U, Lawson A, Ma M, Theobald N, Burkett S, Sweeney CL, Lazzarotto CR, Tsai SQ, Lack JB, Wu X, Dahl GA, Malech HL, Kleinstiver BP, De Ravin SS | Science translational medicine | 39413163 | Sci Transl Med | 2024 Oct 16 |
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| Rapid measurement of hemoglobin-oxygen dissociation by leveraging Bohr effect and Soret band bathochromic shift. | Sekyonda Z, An R, Goreke U, Man Y, Monchamp K, Bode A, Zhang Q, El-Gammal Y, Kityo C, Kalfa TA, Akkus O, Gurkan UA | The Analyst | 38501195 | Analyst | 2024 Apr 29 |
| A miniaturized wash-free microfluidic assay for electrical impedance-based assessment of red blood cell-mediated microvascular occlusion. | Oshabaheebwa S, Delianides CA, Patwardhan AA, Evans EN, Sekyonda Z, Bode A, Apio FM, Mutuluuza CK, Sheehan VA, Suster MA, Gurkan UA, Mohseni P | Biosensors & bioelectronics | 38718635 | Biosens Bioelectron | 2024 Aug 15 |
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| Deleting DNMT3A in CAR T cells prevents exhaustion and enhances antitumor activity. | Prinzing B, Zebley CC, Petersen CT, Fan Y, Anido AA, Yi Z, Nguyen P, Houke H, Bell M, Haydar D, Brown C, Boi SK, Alli S, Crawford JC, Riberdy JM, Park JJ, Zhou S, Velasquez MP, DeRenzo C, Lazzarotto CR, Tsai SQ, Vogel P, Pruett-Miller SM, Langfitt DM, Gottschalk S, Youngblood B, Krenciute G | Science translational medicine | 34788079 | Sci Transl Med | 2021 Nov 17 |
| Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques. | AlJanahi AA, Lazzarotto CR, Chen S, Shin TH, Cordes S, Fan X, Jabara I, Zhou Y, Young DJ, Lee BC, Yu KR, Li Y, Toms B, Tunc I, Hong SG, Truitt LL, Klermund J, Andrieux G, Kim MY, Cathomen T, Gill S, Tsai SQ, Dunbar CE | Molecular therapy : the journal of the American Society of Gene Therapy | 34174439 | Mol Ther | 2022 Jan 5 |
| In vivo engineered B cells secrete high titers of broadly neutralizing anti-HIV antibodies in mice. | Nahmad AD, Lazzarotto CR, Zelikson N, Kustin T, Tenuta M, Huang D, Reuveni I, Nataf D, Raviv Y, Horovitz-Fried M, Dotan I, Carmi Y, Rosin-Arbesfeld R, Nemazee D, Voss JE, Stern A, Tsai SQ, Barzel A | Nature biotechnology | 35681059 | Nat Biotechnol | 2022 Aug |
| CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells. | Brault J, Liu T, Liu S, Lawson A, Choi U, Kozhushko N, Bzhilyanskaya V, Pavel-Dinu M, Meis RJ, Eckhaus MA, Burkett SS, Bosticardo M, Kleinstiver BP, Notarangelo LD, Lazzarotto CR, Tsai SQ, Wu X, Dahl GA, Porteus MH, Malech HL, De Ravin SS | Frontiers in immunology | 36685559 | Front Immunol | 2022 |
| Defining genome-wide CRISPR-Cas genome-editing nuclease activity with GUIDE-seq. | Malinin NL, Lee G, Lazzarotto CR, Li Y, Zheng Z, Nguyen NT, Liebers M, Topkar VV, Iafrate AJ, Le LP, Aryee MJ, Joung JK, Tsai SQ | Nature protocols | 34773119 | Nat Protoc | 2021 Dec |
| Base editing of haematopoietic stem cells rescues sickle cell disease in mice. | Newby GA, Yen JS, Woodard KJ, Mayuranathan T, Lazzarotto CR, Li Y, Sheppard-Tillman H, Porter SN, Yao Y, Mayberry K, Everette KA, Jang Y, Podracky CJ, Thaman E, Lechauve C, Sharma A, Henderson JM, Richter MF, Zhao KT, Miller SM, Wang T, Koblan LW, McCaffrey AP, Tisdale JF, Kalfa TA, Pruett-Miller SM, Tsai SQ, Weiss MJ, Liu DR | Nature | 34079130 | Nature | 2021 Jul |
| Trichostatin A for Efficient CRISPR-Cas9 Gene Editing of Human Pluripotent Stem Cells. | Molugu K, Khajanchi N, Lazzarotto CR, Tsai SQ, Saha K | The CRISPR journal | 37676985 | CRISPR J | 2023 Oct |
| Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression. | Mueller KP, Piscopo NJ, Forsberg MH, Saraspe LA, Das A, Russell B, Smerchansky M, Cappabianca D, Shi L, Shankar K, Sarko L, Khajanchi N, La Vonne Denne N, Ramamurthy A, Ali A, Lazzarotto CR, Tsai SQ, Capitini CM, Saha K | Journal for immunotherapy of cancer | 36382633 | J Immunother Cancer | 2022 Sep |
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| Mechanosensitive genomic enhancers potentiate the cellular response to matrix stiffness. | Cosgrove BD, Bounds LR, Taylor CK, Su AL, Rizzo AJ, Barrera A, Crawford GE, Hoffman BD, Gersbach CA | bioRxiv : the preprint server for biology | 38260455 | bioRxiv | 2024 Jan 10 |
| Activation of the imprinted Prader-Willi Syndrome locus by CRISPR-based epigenome editing. | Rohm D, Black JB, McCutcheon SR, Barrera A, Morone DJ, Nuttle X, de Esch CE, Tai DJC, Talkowski ME, Iglesias N, Gersbach CA | bioRxiv : the preprint server for biology | 38496583 | bioRxiv | 2024 Mar 4 |
| Robust, Durable Gene Activation In Vivo via mRNA-Encoded Activators. | Beyersdorf JP, Bawage S, Iglesias N, Peck HE, Hobbs RA, Wroe JA, Zurla C, Gersbach CA, Santangelo PJ | ACS nano | 35357116 | ACS Nano | 2022 Apr 26 |
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| Master Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation Screens. | Black JB, McCutcheon SR, Dube S, Barrera A, Klann TS, Rice GA, Adkar SS, Soderling SH, Reddy TE, Gersbach CA | Cell reports | 33264623 | Cell Rep | 2020 Dec 1 |
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| Surface Topography of Polyethylene Glycol Shell Nanoparticles Formed from Bottlebrush Block Copolymers Controls Interactions with Proteins and Cells. | Grundler J, Shin K, Suh HW, Zhong M, Saltzman WM | ACS nano | 34633171 | ACS Nano | 2021 Oct 26 |
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| Monobody adapter for functional antibody display on nanoparticles for adaptable targeted delivery applications. | Albert C, Bracaglia L, Koide A, DiRito J, Lysyy T, Harkins L, Edwards C, Richfield O, Grundler J, Zhou K, Denbaum E, Ketavarapu G, Hattori T, Perincheri S, Langford J, Feizi A, Haakinson D, Hosgood SA, Nicholson ML, Pober JS, Saltzman WM, Koide S, Tietjen GT | Nature communications | 36220817 | Nat Commun | 2022 Oct 11 |
| An ELISA-based platform for rapid identification of structure-dependent nucleic acid-protein interactions detects novel DNA triplex interactors. | Economos NG, Thapar U, Balasubramanian N, Karras GI, Glazer PM | The Journal of biological chemistry | 35988651 | J Biol Chem | 2022 Oct |
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| Clades of huge phages from across Earth's ecosystems. | Al-Shayeb B, Sachdeva R, Chen LX, Ward F, Munk P, Devoto A, Castelle CJ, Olm MR, Bouma-Gregson K, Amano Y, He C, Méheust R, Brooks B, Thomas A, Lavy A, Matheus-Carnevali P, Sun C, Goltsman DSA, Borton MA, Sharrar A, Jaffe AL, Nelson TC, Kantor R, Keren R, Lane KR, Farag IF, Lei S, Finstad K, Amundson R, Anantharaman K, Zhou J, Probst AJ, Power ME, Tringe SG, Li WJ, Wrighton K, Harrison S, Morowitz M, Relman DA, Doudna JA, Lehours AC, Warren L, Cate JHD, Santini JM, Banfield JF | Nature | 32051592 | Nature | 2020 Feb |
| Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering. | Hamilton JR, Tsuchida CA, Nguyen DN, Shy BR, McGarrigle ER, Sandoval Espinoza CR, Carr D, Blaeschke F, Marson A, Doudna JA | Cell reports | 34077734 | Cell Rep | 2021 Jun 1 |
| Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cells. | Tsuchida CA, Brandes N, Bueno R, Trinidad M, Mazumder T, Yu B, Hwang B, Chang C, Liu J, Sun Y, Hopkins CR, Parker KR, Qi Y, Satpathy AT, Stadtmauer EA, Cate JHD, Eyquem J, Fraietta JA, June CH, Chang HY, Ye CJ, Doudna JA | bioRxiv : the preprint server for biology | 36993359 | bioRxiv | 2023 Mar 22 |
| In vivo human T cell engineering with enveloped delivery vehicles. | Hamilton JR, Chen E, Perez BS, Sandoval Espinoza CR, Kang MH, Trinidad M, Ngo W, Doudna JA | Nature biotechnology | 38212493 | Nat Biotechnol | 2024 Nov |
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| CRISPR-CasΦ from huge phages is a hypercompact genome editor. | Pausch P, Al-Shayeb B, Bisom-Rapp E, Tsuchida CA, Li Z, Cress BF, Knott GJ, Jacobsen SE, Banfield JF, Doudna JA | Science (New York, N.Y.) | 32675376 | Science | 2020 Jul 17 |
| Eukaryotic RNA-guided endonucleases evolved from a unique clade of bacterial enzymes. | Yoon PH, Skopintsev P, Shi H, Chen L, Adler BA, Al-Shimary M, Craig RJ, Loi KJ, DeTurk EC, Li Z, Amerasekera J, Trinidad M, Nisonoff H, Chen K, Lahiri A, Boger R, Jacobsen S, Banfield JF, Doudna JA | Nucleic acids research | 37971304 | Nucleic Acids Res | 2023 Dec 11 |
| Diverse virus-encoded CRISPR-Cas systems include streamlined genome editors. | Al-Shayeb B, Skopintsev P, Soczek KM, Stahl EC, Li Z, Groover E, Smock D, Eggers AR, Pausch P, Cress BF, Huang CJ, Staskawicz B, Savage DF, Jacobsen SE, Banfield JF, Doudna JA | Cell | 36423580 | Cell | 2022 Nov 23 |
| Precise transcript targeting by CRISPR-Csm complexes. | Colognori D, Trinidad M, Doudna JA | Nature biotechnology | 36690762 | Nat Biotechnol | 2023 Sep |
| Targeted nonviral delivery of genome editors in vivo. | Tsuchida CA, Wasko KM, Hamilton JR, Doudna JA | Proceedings of the National Academy of Sciences of the United States of America | 38437567 | Proc Natl Acad Sci U S A | 2024 Mar 12 |
| The FusX TALE Base Editor (FusXTBE) for Rapid Mitochondrial DNA Programming of Human Cells In Vitro and Zebrafish Disease Models In Vivo. | Sabharwal A, Kar B, Restrepo-Castillo S, Holmberg SR, Mathew ND, Kendall BL, Cotter RP, WareJoncas Z, Seiler C, Nakamaru-Ogiso E, Clark KJ, Ekker SC | The CRISPR journal | 34847747 | CRISPR J | 2021 Dec |
| An optimized FusX assembly-based technique to introduce mitochondrial TC-to-TT variations in human cell lines. | Kar B, Sabharwal A, Restrepo-Castillo S, Simone BW, Clark KJ, Ekker SC | STAR protocols | 35496789 | STAR Protoc | 2022 Jun 17 |
| Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities. | Kar B, Castillo SR, Sabharwal A, Clark KJ, Ekker SC | International journal of molecular sciences | 36982871 | Int J Mol Sci | 2023 Mar 18 |
| Unconstrained Precision Mitochondrial Genome Editing with αDdCBEs. | Castillo SR, Simone BW, Clark KJ, Devaux P, Ekker SC | Human gene therapy | 39212664 | Hum Gene Ther | 2024 Oct |
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| CRISPR-free base editors with enhanced activity and expanded targeting scope in mitochondrial and nuclear DNA. | Mok BY, Kotrys AV, Raguram A, Huang TP, Mootha VK, Liu DR | Nature biotechnology | 35379961 | Nat Biotechnol | 2022 Sep |
| Simultaneous targeting of linked loci in mouse embryos using base editing. | Lee HK, Willi M, Smith HE, Miller SM, Liu DR, Liu C, Hennighausen L | Scientific reports | 30733567 | Sci Rep | 2019 Feb 7 |
| Development of hRad51-Cas9 nickase fusions that mediate HDR without double-stranded breaks. | Rees HA, Yeh WH, Liu DR | Nature communications | 31101808 | Nat Commun | 2019 May 17 |
| Precision genome editing using cytosine and adenine base editors in mammalian cells. | Huang TP, Newby GA, Liu DR | Nature protocols | 33462442 | Nat Protoc | 2021 Feb |
| Phage-assisted continuous and non-continuous evolution. | Miller SM, Wang T, Liu DR | Nature protocols | 33199872 | Nat Protoc | 2020 Dec |
| In vivo HSC prime editing rescues sickle cell disease in a mouse model. | Li C, Georgakopoulou A, Newby GA, Chen PJ, Everette KA, Paschoudi K, Vlachaki E, Gil S, Anderson AK, Koob T, Huang L, Wang H, Kiem HP, Liu DR, Yannaki E, Lieber A | Blood | 36800642 | Blood | 2023 Apr 27 |
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| High-throughput continuous evolution of compact Cas9 variants targeting single-nucleotide-pyrimidine PAMs. | Huang TP, Heins ZJ, Miller SM, Wong BG, Balivada PA, Wang T, Khalil AS, Liu DR | Nature biotechnology | 36076084 | Nat Biotechnol | 2023 Jan |
| Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing. | Pandey S, Gao XD, Krasnow NA, McElroy A, Tao YA, Duby JE, Steinbeck BJ, McCreary J, Pierce SE, Tolar J, Meissner TB, Chaikof EL, Osborn MJ, Liu DR | Nature biomedical engineering | 38858586 | Nat Biomed Eng | 2025 Jan |
| Designing and executing prime editing experiments in mammalian cells. | Doman JL, Sousa AA, Randolph PB, Chen PJ, Liu DR | Nature protocols | 35941224 | Nat Protoc | 2022 Nov |
| Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy. | Kabra M, Shahi PK, Wang Y, Sinha D, Spillane A, Newby GA, Saxena S, Tong Y, Chang Y, Abdeen AA, Edwards KL, Theisen CO, Liu DR, Gamm DM, Gong S, Saha K, Pattnaik BR | The Journal of clinical investigation | 37561581 | J Clin Invest | 2023 Oct 2 |
| Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing. | McAuley GE, Yiu G, Chang PC, Newby GA, Campo-Fernandez B, Fitz-Gibbon ST, Wu X, Kang SL, Garibay A, Butler J, Christian V, Wong RL, Everette KA, Azzun A, Gelfer H, Seet CS, Narendran A, Murguia-Favela L, Romero Z, Wright N, Liu DR, Crooks GM, Kohn DB | Cell | 36944331 | Cell | 2023 Mar 30 |
| Analysis and minimization of cellular RNA editing by DNA adenine base editors. | Rees HA, Wilson C, Doman JL, Liu DR | Science advances | 31086823 | Sci Adv | 2019 May |
| Publisher Correction: Base editing: precision chemistry on the genome and transcriptome of living cells. | Rees HA, Liu DR | Nature reviews. Genetics | 30341440 | Nat Rev Genet | 2018 Dec |
| ABE8e adenine base editor precisely and efficiently corrects a recurrent COL7A1 nonsense mutation. | Sheriff A, Guri I, Zebrowska P, Llopis-Hernandez V, Brooks IR, Tekkela S, Subramaniam K, Gebrezgabher R, Naso G, Petrova A, Balon K, Onoufriadis A, Kujawa D, Kotulska M, Newby G, Łaczmański Ł, Liu DR, McGrath JA, Jacków J | Scientific reports | 36385635 | Sci Rep | 2022 Nov 16 |
| Prioritization of autoimmune disease-associated genetic variants that perturb regulatory element activity in T cells. | Mouri K, Guo MH, de Boer CG, Lissner MM, Harten IA, Newby GA, DeBerg HA, Platt WF, Gentili M, Liu DR, Campbell DJ, Hacohen N, Tewhey R, Ray JP | Nature genetics | 35513721 | Nat Genet | 2022 May |
| PrimeDesign software for rapid and simplified design of prime editing guide RNAs. | Hsu JY, Grünewald J, Szalay R, Shih J, Anzalone AV, Lam KC, Shen MW, Petri K, Liu DR, Joung JK, Pinello L | Nature communications | 33589617 | Nat Commun | 2021 Feb 15 |
| Prime genome editing in rice and wheat. | Lin Q, Zong Y, Xue C, Wang S, Jin S, Zhu Z, Wang Y, Anzalone AV, Raguram A, Doman JL, Liu DR, Gao C | Nature biotechnology | 32393904 | Nat Biotechnol | 2020 May |
| Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity. | Richter MF, Zhao KT, Eton E, Lapinaite A, Newby GA, Thuronyi BW, Wilson C, Koblan LW, Zeng J, Bauer DE, Doudna JA, Liu DR | Nature biotechnology | 32433547 | Nat Biotechnol | 2020 Jul |
| Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors. | Doman JL, Raguram A, Newby GA, Liu DR | Nature biotechnology | 32042165 | Nat Biotechnol | 2020 May |
| A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing. | Mok BY, de Moraes MH, Zeng J, Bosch DE, Kotrys AV, Raguram A, Hsu F, Radey MC, Peterson SB, Mootha VK, Mougous JD, Liu DR | Nature | 32641830 | Nature | 2020 Jul |
| Disruption of HIV-1 co-receptors CCR5 and CXCR4 in primary human T cells and hematopoietic stem and progenitor cells using base editing. | Knipping F, Newby GA, Eide CR, McElroy AN, Nielsen SC, Smith K, Fang Y, Cornu TI, Costa C, Gutierrez-Guerrero A, Bingea SP, Feser CJ, Steinbeck B, Hippen KL, Blazar BR, McCaffrey A, Mussolino C, Verhoeyen E, Tolar J, Liu DR, Osborn MJ | Molecular therapy : the journal of the American Society of Gene Therapy | 34737067 | Mol Ther | 2022 Jan 5 |
| Engineered pegRNAs improve prime editing efficiency. | Nelson JW, Randolph PB, Shen SP, Everette KA, Chen PJ, Anzalone AV, An M, Newby GA, Chen JC, Hsu A, Liu DR | Nature biotechnology | 34608327 | Nat Biotechnol | 2022 Mar |
| Enhanced prime editing systems by manipulating cellular determinants of editing outcomes. | Chen PJ, Hussmann JA, Yan J, Knipping F, Ravisankar P, Chen PF, Chen C, Nelson JW, Newby GA, Sahin M, Osborn MJ, Weissman JS, Adamson B, Liu DR | Cell | 34653350 | Cell | 2021 Oct 28 |
| Massively parallel base editing to map variant effects in human hematopoiesis. | Martin-Rufino JD, Castano N, Pang M, Grody EI, Joubran S, Caulier A, Wahlster L, Li T, Qiu X, Riera-Escandell AM, Newby GA, Al'Khafaji A, Chaudhary S, Black S, Weng C, Munson G, Liu DR, Wlodarski MW, Sims K, Oakley JH, Fasano RM, Xavier RJ, Lander ES, Klein DE, Sankaran VG | Cell | 37137305 | Cell | 2023 May 25 |
| A prime editor mouse to model a broad spectrum of somatic mutations in vivo. | Ely ZA, Mathey-Andrews N, Naranjo S, Gould SI, Mercer KL, Newby GA, Cabana CM, Rideout WM 3rd, Jaramillo GC, Khirallah JM, Holland K, Randolph PB, Freed-Pastor WA, Davis JR, Kulstad Z, Westcott PMK, Lin L, Anzalone AV, Horton BL, Pattada NB, Shanahan SL, Ye Z, Spranger S, Xu Q, Sánchez-Rivera FJ, Liu DR, Jacks T | Nature biotechnology | 37169967 | Nat Biotechnol | 2024 Mar |
| Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice. | Everette KA, Newby GA, Levine RM, Mayberry K, Jang Y, Mayuranathan T, Nimmagadda N, Dempsey E, Li Y, Bhoopalan SV, Liu X, Davis JR, Nelson AT, Chen PJ, Sousa AA, Cheng Y, Tisdale JF, Weiss MJ, Yen JS, Liu DR | Nature biomedical engineering | 37069266 | Nat Biomed Eng | 2023 May |
| Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice. | Reichart D, Newby GA, Wakimoto H, Lun M, Gorham JM, Curran JJ, Raguram A, DeLaughter DM, Conner DA, Marsiglia JDC, Kohli S, Chmatal L, Page DC, Zabaleta N, Vandenberghe L, Liu DR, Seidman JG, Seidman C | Nature medicine | 36797483 | Nat Med | 2023 Feb |
| Compact zinc finger base editors that edit mitochondrial or nuclear DNA in vitro and in vivo. | Willis JCW, Silva-Pinheiro P, Widdup L, Minczuk M, Liu DR | Nature communications | 36418298 | Nat Commun | 2022 Nov 23 |
| Efficient prime editing in two-cell mouse embryos using PEmbryo. | Kim-Yip RP, McNulty R, Joyce B, Mollica A, Chen PJ, Ravisankar P, Law BK, Liu DR, Toettcher JE, Ivakine EA, Posfai E, Adamson B | Nature biotechnology | 38321114 | Nat Biotechnol | 2024 Dec |
| Disulfide-compatible phage-assisted continuous evolution in the periplasmic space. | Morrison MS, Wang T, Raguram A, Hemez C, Liu DR | Nature communications | 34645844 | Nat Commun | 2021 Oct 13 |
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| An anionic human protein mediates cationic liposome delivery of genome editing proteins into mammalian cells. | Kim YB, Zhao KT, Thompson DB, Liu DR | Nature communications | 31266953 | Nat Commun | 2019 Jul 2 |
| Continuous evolution of base editors with expanded target compatibility and improved activity. | Thuronyi BW, Koblan LW, Levy JM, Yeh WH, Zheng C, Newby GA, Wilson C, Bhaumik M, Shubina-Oleinik O, Holt JR, Liu DR | Nature biotechnology | 31332326 | Nat Biotechnol | 2019 Sep |
| Mechanisms of angiogenic incompetence in Hutchinson-Gilford progeria via downregulation of endothelial NOS. | Gete YG, Koblan LW, Mao X, Trappio M, Mahadik B, Fisher JP, Liu DR, Cao K | Aging cell | 34086398 | Aging Cell | 2021 Jul |
| Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity. | Neugebauer ME, Hsu A, Arbab M, Krasnow NA, McElroy AN, Pandey S, Doman JL, Huang TP, Raguram A, Banskota S, Newby GA, Tolar J, Osborn MJ, Liu DR | Nature biotechnology | 36357719 | Nat Biotechnol | 2023 May |
| In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies. | Li C, Georgakopoulou A, Newby GA, Everette KA, Nizamis E, Paschoudi K, Vlachaki E, Gil S, Anderson AK, Koob T, Huang L, Wang H, Kiem HP, Liu DR, Yannaki E, Lieber A | JCI insight | 36006707 | JCI Insight | 2022 Oct 10 |
| Assigning functionality to cysteines by base editing of cancer dependency genes. | Li H, Ma T, Remsberg JR, Won SJ, DeMeester KE, Njomen E, Ogasawara D, Zhao KT, Huang TP, Lu B, Simon GM, Melillo B, Schreiber SL, Lykke-Andersen J, Liu DR, Cravatt BF | Nature chemical biology | 37783940 | Nat Chem Biol | 2023 Nov |
| Precision genome editing in the eye. | Suh S, Choi EH, Raguram A, Liu DR, Palczewski K | Proceedings of the National Academy of Sciences of the United States of America | 36122230 | Proc Natl Acad Sci U S A | 2022 Sep 27 |
| The developing toolkit of continuous directed evolution. | Morrison MS, Podracky CJ, Liu DR | Nature chemical biology | 32444838 | Nat Chem Biol | 2020 Jun |
| Search-and-replace genome editing without double-strand breaks or donor DNA. | Anzalone AV, Randolph PB, Davis JR, Sousa AA, Koblan LW, Levy JM, Chen PJ, Wilson C, Newby GA, Raguram A, Liu DR | Nature | 31634902 | Nature | 2019 Dec |
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| Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs. | Osborn MJ, Newby GA, McElroy AN, Knipping F, Nielsen SC, Riddle MJ, Xia L, Chen W, Eide CR, Webber BR, Wandall HH, Dabelsteen S, Blazar BR, Liu DR, Tolar J | The Journal of investigative dermatology | 31437443 | J Invest Dermatol | 2020 Feb |
| Programmable m(6)A modification of cellular RNAs with a Cas13-directed methyltransferase. | Wilson C, Chen PJ, Miao Z, Liu DR | Nature biotechnology | 32601430 | Nat Biotechnol | 2020 Dec |
| Continuous evolution of SpCas9 variants compatible with non-G PAMs. | Miller SM, Wang T, Randolph PB, Arbab M, Shen MW, Huang TP, Matuszek Z, Newby GA, Rees HA, Liu DR | Nature biotechnology | 32042170 | Nat Biotechnol | 2020 Apr |
| Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing. | Anzalone AV, Gao XD, Podracky CJ, Nelson AT, Koblan LW, Raguram A, Levy JM, Mercer JAM, Liu DR | Nature biotechnology | 34887556 | Nat Biotechnol | 2022 May |
| Base editing rescue of spinal muscular atrophy in cells and in mice. | Arbab M, Matuszek Z, Kray KM, Du A, Newby GA, Blatnik AJ, Raguram A, Richter MF, Zhao KT, Levy JM, Shen MW, Arnold WD, Wang D, Xie J, Gao G, Burghes AHM, Liu DR | Science (New York, N.Y.) | 36996170 | Science | 2023 Apr 21 |
| Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells. | Joynt AT, Kavanagh EW, Newby GA, Mitchell S, Eastman AC, Paul KC, Bowling AD, Osorio DL, Merlo CA, Patel SU, Raraigh KS, Liu DR, Sharma N, Cutting GR | Molecular therapy. Nucleic acids | 37547293 | Mol Ther Nucleic Acids | 2023 Sep 12 |
| Branched chemically modified poly(A) tails enhance the translation capacity of mRNA. | Chen H, Liu D, Guo J, Aditham A, Zhou Y, Tian J, Luo S, Ren J, Hsu A, Huang J, Kostas F, Wu M, Liu DR, Wang X | Nature biotechnology | 38519719 | Nat Biotechnol | 2025 Feb |
