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2022 1
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Page 1
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. Li C, et al. Blood. 2023 Apr 27;141(17):2085-2099. doi: 10.1182/blood.2022018252. Blood. 2023. PMID: 36800642 Free PMC article.
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. Reichart D, et al. Nat Med. 2023 Feb;29(2):412-421. doi: 10.1038/s41591-022-02190-7. Epub 2023 Feb 16. Nat Med. 2023. PMID: 36797483 Free PMC article.
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. Arbab M, et al. Science. 2023 Apr 21;380(6642):eadg6518. doi: 10.1126/science.adg6518. Epub 2023 Apr 14. Science. 2023. PMID: 36996170 Free PMC article.
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. McAuley GE, et al. Cell. 2023 Mar 30;186(7):1398-1416.e23. doi: 10.1016/j.cell.2023.02.027. Epub 2023 Mar 20. Cell. 2023. PMID: 36944331 Free PMC article.
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. Mayuranathan T, et al. Nat Genet. 2023 Jul;55(7):1210-1220. doi: 10.1038/s41588-023-01434-7. Epub 2023 Jul 3. Nat Genet. 2023. PMID: 37400614 Free PMC article.
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. Davis JR, et al. Nat Biotechnol. 2024 Feb;42(2):253-264. doi: 10.1038/s41587-023-01758-z. Epub 2023 May 4. Nat Biotechnol. 2024. PMID: 37142705 Free PMC article.
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. Ely ZA, et al. Nat Biotechnol. 2024 Mar;42(3):424-436. doi: 10.1038/s41587-023-01783-y. Epub 2023 May 11. Nat Biotechnol. 2024. PMID: 37169967 Free PMC article.
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. Kulhankova K, et al. Nat Commun. 2023 Dec 5;14(1):8051. doi: 10.1038/s41467-023-43904-w. Nat Commun. 2023. PMID: 38052872 Free PMC article.
13 results