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2024

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

A benchmarked, high-efficiency prime editing platform for multiplexed dropout screening

Cirincione A, Simpson D, Ravisankar P, Solley SC, Yan J, Singh M, Adamson B

Improving prime editing with an endogenous small RNA-binding protein

Yan J, Oyler-Castrillo P, Ravisankar P, Ward CC, Levesque S, Jing Y, Simpson D, Zhao A, Li H, Yan W, Goudy L, Schmidt R, Solley SC, Gilbert LA, Chan MM, Bauer DE, Marson A, Parsons LR, Adamson B

Efficient prime editing in mouse brain, liver and heart with dual AAVs

Davis JR, Banskota S, Levy JM, Newby GA, Wang X, Anzalone AV, Nelson AT, Chen PJ, Hennes AD, An M, Roh H, Randolph PB, Musunuru K, Liu DR

2023

Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo

Kulhankova K, Traore S, Cheng X, Benk-Fortin H, Hallée S, Harvey M, Roberge J, Couture F, Kohli S, Gross TJ, Meyerholz DK, Rettig GR, Thommandru B, Kurgan G, Wohlford-Lenane C, Hartigan-O'Connor DJ, Yates BP, Newby GA, Liu DR, Tarantal AF, Guay D, McCray PB Jr

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

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

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

Phage-assisted evolution and protein engineering yield compact, efficient prime editors

Doman JL, Pandey S, Neugebauer ME, An M, Davis JR, Randolph PB, McElroy A, Gao XD, Raguram A, Richter MF, Everette KA, Banskota S, Tian K, Tao YA, Tolar J, Osborn MJ, Liu DR

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

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

Potent and uniform fetal hemoglobin induction via base editing

Mayuranathan T, Newby GA, Feng R, Yao Y, Mayberry KD, Lazzarotto CR, Li Y, Levine RM, Nimmagadda N, Dempsey E, Kang G, Porter SN, Doerfler PA, Zhang J, Jang Y, Chen J, Bell HW, Crossley M, Bhoopalan SV, Sharma A, Tisdale JF, Pruett-Miller SM, Cheng Y, Tsai SQ, Liu DR, Weiss MJ, Yen JS

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

Massively parallel base editing to map variant effects in human hematopoiesis

DR, Wlodarski MW, Sims K, Oakley JH, Fasano RM, Xavier RJ, Lander ES, Klein DE, Sankaran VG

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

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

Human genetic diversity alters off-target outcomes of therapeutic gene editing

Cancellieri S, Zeng J, Lin LY, Tognon M, Nguyen MA, Lin J, Bombieri N, Maitland SA, Ciuculescu MF, Katta V, Tsai SQ, Armant M, Wolfe SA, Giugno R, Bauer DE, Pinello L

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

2022

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

MTCH2 is a mitochondrial outer membrane protein insertase

Guna A, Stevens TA, Inglis AJ, Replogle JM, Esantsi TK, Muthukumar G, Shaffer KCL, Wang ML, Pogson AN, Jones JJ, Lomenick B, Chou TF, Weissman JS, Voorhees RM

Lineage tracing reveals the phylodynamics, plasticity, and paths of tumor evolution

Yang D, Jones MG, Naranjo S, Rideout WM 3rd, Min KHJ, Ho R, Wu W, Replogle JM, Page JL, Quinn JJ, Horns F, Qiu X, Chen MZ, Freed-Pastor WA, McGinnis CS, Patterson DM, Gartner ZJ, Chow ED, Bivona TG, Chan MM, Yosef N, Jacks T, Weissman JS

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

Designing and executing prime editing experiments in mammalian cells

Doman JL, Sousa AA, Randolph PB, Chen PJ, Liu DR

Precision genome editing in the eye

Suh S, Choi EH, Raguram A, Liu DR, Palczewski K

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

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

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

In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration

Choi EH, Suh S, Foik AT, Leinonen H, Newby GA, Gao XD, Banskota S, Hoang T, Du SW, Dong Z, Raguram A, Kohli S, Blackshaw S, Lyon DC, Liu DR, Palczewski K

Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins

Banskota S, Raguram A, Suh S, Du SW, Davis JR, Choi EH, Wang X, Nielsen SC, Newby GA, Randolph PB, Osborn MJ, Musunuru K, Palczewski K, Liu DR