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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: sevin c. 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.
Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
Bey K, Deniaud J, Dubreil L, Joussemet B, Cristini J, Ciron C, Hordeaux J, Le Boulc'h M, Marche K, Maquigneau M, Guilbaud M, Moreau R, Larcher T, Deschamps JY, Fusellier M, Blouin V, Sevin C, Cartier N, Adjali O, Aubourg P, Moullier P, Colle MA. Bey K, et al. Among authors: sevin c. Mol Ther Methods Clin Dev. 2020 Apr 11;17:771-784. doi: 10.1016/j.omtm.2020.04.001. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32355866 Free PMC article.
Gene therapy in metachromatic leukodystrophy.
Sevin C, Cartier-Lacave N, Aubourg P. Sevin C, et al. Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S128-31. doi: 10.5414/cpp47128. Int J Clin Pharmacol Ther. 2009. PMID: 20040324 Review.
128 results