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Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.
Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U, Phillips M, Riddell A, Peralta MR, Quaye M, Patch DW, Tuddenham E, Dane A, Watissée M, Long A, Nathwani A. Chowdary P, et al. Among authors: nathwani a. N Engl J Med. 2022 Jul 21;387(3):237-247. doi: 10.1056/NEJMoa2119913. N Engl J Med. 2022. PMID: 35857660 Free article. Clinical Trial.
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: nathwani a. 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.
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease.
Jeyakumar JM, Kia A, Tam LCS, McIntosh J, Spiewak J, Mills K, Heywood W, Chisari E, Castaldo N, Verhoef D, Hosseini P, Kalcheva P, Cocita C, Miranda CJ, Canavese M, Khinder J, Rosales C, Hughes D, Sheridan R, Corbau R, Nathwani A. Jeyakumar JM, et al. Among authors: nathwani a. Gene Ther. 2023 Jun;30(6):487-502. doi: 10.1038/s41434-022-00381-y. Epub 2023 Jan 11. Gene Ther. 2023. PMID: 36631545 Free PMC article.
The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins.
Paul-Smith MC, Pytel KM, Gelinas JF, McIntosh J, Pringle I, Davies L, Chan M, Meng C, Bell R, Cammack L, Moran C, Cameron L, Inoue M, Tsugumine S, Hironaka T, Gill DR, Hyde SC, Nathwani A, Alton EWFW, Griesenbach U. Paul-Smith MC, et al. Among authors: nathwani a. Gene Ther. 2018 Aug;25(5):345-358. doi: 10.1038/s41434-018-0025-8. Epub 2018 Jul 18. Gene Ther. 2018. PMID: 30022127 Free PMC article.
171 results