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Page 1
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques.
Blackwood M, Gruntman AM, Tang Q, Pires-Ferreira D, Reil D, Kondratov O, Marsic D, Zolotukhin S, Gernoux G, Keeler AM, Mueller C, Flotte TR. Blackwood M, et al. Among authors: gernoux g. Mol Ther Methods Clin Dev. 2024 Jan 30;32(1):101200. doi: 10.1016/j.omtm.2024.101200. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38445045 Free PMC article.
AAV gene therapy for Tay-Sachs disease.
Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, Douthwright C, Gernoux G, Blackwood M, Mueller C, Tai PWL, Jiang X, Bateman S, Spanakis SG, Parzych J, Keeler AM, Abayazeed A, Rohatgi S, Gibson L, Finberg R, Barton BA, Vardar Z, Shazeeb MS, Gounis M, Tifft CJ, Eichler FS, Brown RH Jr, Martin DR, Gray-Edwards HL, Sena-Esteves M. Flotte TR, et al. Among authors: gernoux g. Nat Med. 2022 Feb;28(2):251-259. doi: 10.1038/s41591-021-01664-4. Epub 2022 Feb 10. Nat Med. 2022. PMID: 35145305 Free PMC article.
AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation.
Gernoux G, Guilbaud M, Devaux M, Journou M, Pichard V, Jaulin N, Léger A, Le Duff J, Deschamps JY, Le Guiner C, Moullier P, Cherel Y, Adjali O. Gernoux G, et al. Mol Ther Methods Clin Dev. 2021 Feb 6;20:660-674. doi: 10.1016/j.omtm.2021.02.003. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2021. PMID: 33718516 Free PMC article.
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.
Mueller C, Berry JD, McKenna-Yasek DM, Gernoux G, Owegi MA, Pothier LM, Douthwright CL, Gelevski D, Luppino SD, Blackwood M, Wightman NS, Oakley DH, Frosch MP, Flotte TR, Cudkowicz ME, Brown RH Jr. Mueller C, et al. Among authors: gernoux g. N Engl J Med. 2020 Jul 9;383(2):151-158. doi: 10.1056/NEJMoa2005056. N Engl J Med. 2020. PMID: 32640133
22 results