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-Reference-Cited by-同舟云学术

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

Published:2015-06 Issue:2 Volume:26 Page:113-124
ISSN:2324-8637
Container-title:Human Gene Therapy Clinical Development
language:en
Short-container-title:Human Gene Therapy Clinical Development

Author:

Zerah Michel¹²,Piguet Françoise¹,Colle Marie-Anne³⁴,Raoul Sylvie⁵,Deschamps Jack-Yves³⁴,Deniaud Johan³,Gautier Benoit¹,Toulgoat Frédérique⁶,Bieche Ivan⁷,Laurendeau Ingrid⁷,Sondhi Dolan⁸,Souweidane Mark M.⁹,Cartier-Lacave Nathalie¹,Moullier Philippe¹⁰,Crystal Ronald G.⁸,Roujeau Thomas¹¹,Sevin Caroline¹¹²,Aubourg Patrick¹¹²

Affiliation:

1. Inserm U986, 94275 Le Kremlin Bicêtre, France.

2. Pediatric Neurosurgery, Necker Children's Hospital, 75014 Paris, France.

3. INRA UMR U703, 44000 Nantes, France.

4. Food Science and Engineering Oniris, Nantes-Atlantic College of Veterinary Medicine, 44000 Nantes, France.

5. Service de Neurochirurgie, CHU Nord, 44000 Nantes, France.

6. Neuroradiologie Diagnostique et Interventionnelle, Hôpital Laennec, CHU de Nantes, 44000 Nantes, France.

7. Faculté des Sciences Pharmaceutiques et Biologiques, 75005 Paris, France.

8. Department of Genetic Medicine, Weill-Cornell Medical College, New York, NY 10065.

9. Neurological Surgery and Pediatrics, Weill-Cornell Medical College, New York, NY 10065.

10. Atlantic Gene Therapies, INSERM UMR 1089, 44000 Nantes, France.

11. Neurosurgery, Hôpitaux de Montpellier, 34000 Montpellier, France.