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Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy.
Lek A, Wong B, Keeler A, Blackwood M, Ma K, Huang S, Sylvia K, Batista AR, Artinian R, Kokoski D, Parajuli S, Putra J, Carreon CK, Lidov H, Woodman K, Pajusalu S, Spinazzola JM, Gallagher T, LaRovere J, Balderson D, Black L, Sutton K, Horgan R, Lek M, Flotte T. Lek A, et al. N Engl J Med. 2023 Sep 28;389(13):1203-1210. doi: 10.1056/NEJMoa2307798. N Engl J Med. 2023. PMID: 37754285 Free PMC article.
Direct ITR-to-ITR Nanopore Sequencing of AAV Vector Genomes.
Namkung S, Tran NT, Manokaran S, He R, Su Q, Xie J, Gao G, Tai PWL. Namkung S, et al. Hum Gene Ther. 2022 Nov;33(21-22):1187-1196. doi: 10.1089/hum.2022.143. Hum Gene Ther. 2022. PMID: 36178359 Free PMC article.
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice.
Wang J, Zhang Y, Mendonca CA, Yukselen O, Muneeruddin K, Ren L, Liang J, Zhou C, Xie J, Li J, Jiang Z, Kucukural A, Shaffer SA, Gao G, Wang D. Wang J, et al. Nature. 2022 Apr;604(7905):343-348. doi: 10.1038/s41586-022-04533-3. Epub 2022 Mar 23. Nature. 2022. PMID: 35322228 Free PMC article.
Durability of transgene expression after rAAV gene therapy.
Muhuri M, Levy DI, Schulz M, McCarty D, Gao G. Muhuri M, et al. Mol Ther. 2022 Apr 6;30(4):1364-1380. doi: 10.1016/j.ymthe.2022.03.004. Epub 2022 Mar 10. Mol Ther. 2022. PMID: 35283274 Free PMC article. Review.
24 results