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Temporary Reduction of Membrane CD4 with the Antioxidant MnTBAP Is Sufficient to Prevent Immune Responses Induced by Gene Transfer.
Da Rocha S, Bigot J, Onodi F, Cosette J, Corre G, Poupiot J, Fenard D, Gjata B, Galy A, Neildez-Nguyen TMA. Da Rocha S, et al. Among authors: gjata b. Mol Ther Methods Clin Dev. 2019 Jul 23;14:285-299. doi: 10.1016/j.omtm.2019.06.011. eCollection 2019 Sep 13. Mol Ther Methods Clin Dev. 2019. PMID: 31497619 Free PMC article.
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.
Vidal P, Pagliarani S, Colella P, Costa Verdera H, Jauze L, Gjorgjieva M, Puzzo F, Marmier S, Collaud F, Simon Sola M, Charles S, Lucchiari S, van Wittenberghe L, Vignaud A, Gjata B, Richard I, Laforet P, Malfatti E, Mithieux G, Rajas F, Comi GP, Ronzitti G, Mingozzi F. Vidal P, et al. Among authors: gjata b. Mol Ther. 2018 Mar 7;26(3):890-901. doi: 10.1016/j.ymthe.2017.12.019. Epub 2017 Dec 28. Mol Ther. 2018. PMID: 29396266 Free PMC article.
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.
Le Guiner C, Servais L, Montus M, Larcher T, Fraysse B, Moullec S, Allais M, François V, Dutilleul M, Malerba A, Koo T, Thibaut JL, Matot B, Devaux M, Le Duff J, Deschamps JY, Barthelemy I, Blot S, Testault I, Wahbi K, Ederhy S, Martin S, Veron P, Georger C, Athanasopoulos T, Masurier C, Mingozzi F, Carlier P, Gjata B, Hogrel JY, Adjali O, Mavilio F, Voit T, Moullier P, Dickson G. Le Guiner C, et al. Among authors: gjata b. Nat Commun. 2017 Jul 25;8:16105. doi: 10.1038/ncomms16105. Nat Commun. 2017. PMID: 28742067 Free PMC article.
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.
Armbruster N, Lattanzi A, Jeavons M, Van Wittenberghe L, Gjata B, Marais T, Martin S, Vignaud A, Voit T, Mavilio F, Barkats M, Buj-Bello A. Armbruster N, et al. Among authors: gjata b. Mol Ther Methods Clin Dev. 2016 Sep 14;3:16060. doi: 10.1038/mtm.2016.60. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27652289 Free PMC article.
AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice.
Colella P, Sellier P, Costa Verdera H, Puzzo F, van Wittenberghe L, Guerchet N, Daniele N, Gjata B, Marmier S, Charles S, Simon Sola M, Ragone I, Leborgne C, Collaud F, Mingozzi F. Colella P, et al. Among authors: gjata b. Mol Ther Methods Clin Dev. 2018 Nov 17;12:85-101. doi: 10.1016/j.omtm.2018.11.002. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30581888 Free PMC article.
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
Costa-Verdera H, Collaud F, Riling CR, Sellier P, Nordin JML, Preston GM, Cagin U, Fabregue J, Barral S, Moya-Nilges M, Krijnse-Locker J, van Wittenberghe L, Daniele N, Gjata B, Cosette J, Abad C, Simon-Sola M, Charles S, Li M, Crosariol M, Antrilli T, Quinn WJ 3rd, Gross DA, Boyer O, Anguela XM, Armour SM, Colella P, Ronzitti G, Mingozzi F. Costa-Verdera H, et al. Among authors: gjata b. Nat Commun. 2021 Nov 4;12(1):6393. doi: 10.1038/s41467-021-26744-4. Nat Commun. 2021. PMID: 34737297 Free PMC article.
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.
Ronzitti G, Bortolussi G, van Dijk R, Collaud F, Charles S, Leborgne C, Vidal P, Martin S, Gjata B, Sola MS, van Wittenberghe L, Vignaud A, Veron P, Bosma PJ, Muro AF, Mingozzi F. Ronzitti G, et al. Among authors: gjata b. Mol Ther Methods Clin Dev. 2016 Jul 20;3:16049. doi: 10.1038/mtm.2016.49. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27722180 Free PMC article.
27 results