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Page 1
Gene therapy in metachromatic leukodystrophy.
Sevin C, Cartier-Lacave N, Aubourg P. Sevin C, et al. Among authors: cartier lacave n. Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S128-31. doi: 10.5414/cpp47128. Int J Clin Pharmacol Ther. 2009. PMID: 20040324 Review.
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: cartier lacave n. 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.
Debate on Germline Gene Editing.
Cartier-Lacave N, Ali R, Ylä-Herttuala S, Kato K, Baetschi B, Lovell-Badge R, Naldini L, Thrasher A. Cartier-Lacave N, et al. Hum Gene Ther Methods. 2016 Aug;27(4):135-42. doi: 10.1089/hgtb.2016.28999.deb. Hum Gene Ther Methods. 2016. PMID: 27529507 No abstract available.