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Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates.
Zerah M, Piguet F, Colle MA, Raoul S, Deschamps JY, Deniaud J, Gautier B, Toulgoat F, Bieche I, Laurendeau I, Sondhi D, Souweidane MM, Cartier-Lacave N, Moullier P, Crystal RG, Roujeau T, Sevin C, Aubourg P. Zerah M, et al. Among authors: piguet f. Hum Gene Ther Clin Dev. 2015 Jun;26(2):113-24. doi: 10.1089/humc.2014.139. Epub 2015 Apr 28. Hum Gene Ther Clin Dev. 2015. PMID: 25758611 Clinical Trial.
Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain.
Orefice NS, Souchet B, Braudeau J, Alves S, Piguet F, Collaud F, Ronzitti G, Tada S, Hantraye P, Mingozzi F, Ducongé F, Cartier N. Orefice NS, et al. Among authors: piguet f. Mol Ther Methods Clin Dev. 2019 Jul 3;14:237-251. doi: 10.1016/j.omtm.2019.06.005. eCollection 2019 Sep 13. Mol Ther Methods Clin Dev. 2019. PMID: 31440523 Free PMC article.
40 results