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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. Saha K, et al. Among authors: bankiewicz ks. Nature. 2021 Apr;592(7853):195-204. doi: 10.1038/s41586-021-03191-1. Epub 2021 Apr 7. Nature. 2021. PMID: 33828315 Free PMC article. Review.
Gene therapy for neurological disorders: progress and prospects.
Deverman BE, Ravina BM, Bankiewicz KS, Paul SM, Sah DWY. Deverman BE, et al. Among authors: bankiewicz ks. Nat Rev Drug Discov. 2018 Sep;17(9):641-659. doi: 10.1038/nrd.2018.110. Epub 2018 Aug 10. Nat Rev Drug Discov. 2018. PMID: 30093643 Review.
Gene therapy for neurological disorders: progress and prospects.
Deverman BE, Ravina BM, Bankiewicz KS, Paul SM, Sah DWY. Deverman BE, et al. Among authors: bankiewicz ks. Nat Rev Drug Discov. 2018 Oct;17(10):767. doi: 10.1038/nrd.2018.158. Epub 2018 Sep 12. Nat Rev Drug Discov. 2018. PMID: 30206384
Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.
Pearson TS, Gupta N, San Sebastian W, Imamura-Ching J, Viehoever A, Grijalvo-Perez A, Fay AJ, Seth N, Lundy SM, Seo Y, Pampaloni M, Hyland K, Smith E, de Oliveira Barbosa G, Heathcock JC, Minnema A, Lonser R, Elder JB, Leonard J, Larson P, Bankiewicz KS. Pearson TS, et al. Among authors: bankiewicz ks. Nat Commun. 2021 Jul 12;12(1):4251. doi: 10.1038/s41467-021-24524-8. Nat Commun. 2021. PMID: 34253733 Free PMC article. Clinical Trial.
GDNF gene therapy for alcohol use disorder in male non-human primates.
Ford MM, George BE, Van Laar VS, Holleran KM, Naidoo J, Hadaczek P, Vanderhooft LE, Peck EG, Dawes MH, Ohno K, Bringas J, McBride JL, Samaranch L, Forsayeth JR, Jones SR, Grant KA, Bankiewicz KS. Ford MM, et al. Among authors: bankiewicz ks. Nat Med. 2023 Aug;29(8):2030-2040. doi: 10.1038/s41591-023-02463-9. Epub 2023 Aug 14. Nat Med. 2023. PMID: 37580533 Free PMC article.
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