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Restoration of visual function in advanced disease after transplantation of purified human pluripotent stem cell-derived cone photoreceptors.
Ribeiro J, Procyk CA, West EL, O'Hara-Wright M, Martins MF, Khorasani MM, Hare A, Basche M, Fernando M, Goh D, Jumbo N, Rizzi M, Powell K, Tariq M, Michaelides M, Bainbridge JWB, Smith AJ, Pearson RA, Gonzalez-Cordero A, Ali RR. Ribeiro J, et al. Among authors: basche m. Cell Rep. 2021 Apr 20;35(3):109022. doi: 10.1016/j.celrep.2021.109022. Cell Rep. 2021. PMID: 33882303 Free PMC article.
Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors.
Tan MH, Smith AJ, Pawlyk B, Xu X, Liu X, Bainbridge JB, Basche M, McIntosh J, Tran HV, Nathwani A, Li T, Ali RR. Tan MH, et al. Among authors: basche m. Hum Mol Genet. 2009 Jun 15;18(12):2099-114. doi: 10.1093/hmg/ddp133. Epub 2009 Mar 19. Hum Mol Genet. 2009. PMID: 19299492 Free PMC article.
A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.
Tordo J, O'Leary C, Antunes ASLM, Palomar N, Aldrin-Kirk P, Basche M, Bennett A, D'Souza Z, Gleitz H, Godwin A, Holley RJ, Parker H, Liao AY, Rouse P, Youshani AS, Dridi L, Martins C, Levade T, Stacey KB, Davis DM, Dyer A, Clément N, Björklund T, Ali RR, Agbandje-McKenna M, Rahim AA, Pshezhetsky A, Waddington SN, Linden RM, Bigger BW, Henckaerts E. Tordo J, et al. Among authors: basche m. Brain. 2018 Jul 1;141(7):2014-2031. doi: 10.1093/brain/awy126. Brain. 2018. PMID: 29788236 Free PMC article.
28 results