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Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants.
Weinmann J, Weis S, Sippel J, Tulalamba W, Remes A, El Andari J, Herrmann AK, Pham QH, Borowski C, Hille S, Schönberger T, Frey N, Lenter M, VandenDriessche T, Müller OJ, Chuah MK, Lamla T, Grimm D. Weinmann J, et al. Among authors: pham qh. Nat Commun. 2020 Oct 28;11(1):5432. doi: 10.1038/s41467-020-19230-w. Nat Commun. 2020. PMID: 33116134 Free PMC article.
Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders.
El Andari J, Renaud-Gabardos E, Tulalamba W, Weinmann J, Mangin L, Pham QH, Hille S, Bennett A, Attebi E, Bourges E, Leborgne C, Guerchet N, Fakhiri J, Krämer C, Wiedtke E, McKenna R, Guianvarc'h L, Toueille M, Ronzitti G, Hebben M, Mingozzi F, VandenDriessche T, Agbandje-McKenna M, Müller OJ, Chuah MK, Buj-Bello A, Grimm D. El Andari J, et al. Among authors: pham qh. Sci Adv. 2022 Sep 23;8(38):eabn4704. doi: 10.1126/sciadv.abn4704. Epub 2022 Sep 21. Sci Adv. 2022. PMID: 36129972 Free PMC article.
Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy.
Muñoz S, Bertolin J, Jimenez V, Jaén ML, Garcia M, Pujol A, Vilà L, Sacristan V, Barbon E, Ronzitti G, El Andari J, Tulalamba W, Pham QH, Ruberte J, VandenDriessche T, Chuah MK, Grimm D, Mingozzi F, Bosch F. Muñoz S, et al. Among authors: pham qh. Mol Metab. 2024 Mar;81:101899. doi: 10.1016/j.molmet.2024.101899. Epub 2024 Feb 10. Mol Metab. 2024. PMID: 38346589 Free PMC article.
20 results