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Gene therapy in metachromatic leukodystrophy.
Sevin C, Cartier-Lacave N, Aubourg P. Sevin C, et al. Among authors: aubourg p. 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: aubourg p. 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.
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Eichler F, et al. Among authors: aubourg p. N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4. N Engl J Med. 2017. PMID: 28976817 Free PMC article. Clinical Trial.
Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates.
Rosenberg JB, Sondhi D, Rubin DG, Monette S, Chen A, Cram S, De BP, Kaminsky SM, Sevin C, Aubourg P, Crystal RG. Rosenberg JB, et al. Among authors: aubourg p. Hum Gene Ther Clin Dev. 2014 Sep;25(3):164-77. doi: 10.1089/humc.2013.239. Epub 2014 Aug 21. Hum Gene Ther Clin Dev. 2014. PMID: 25144894 Free PMC article.
182 results