Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.
Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by providing a durable therapeutic protein via a single administration. Adeno-associated virus (AAV)-mediated gene transfer is of particular interest as a therapeutic tool because of its safety profile and efficiency in transducing a wide range of cell types. The purpose of this review is to describe the most notable advancements in preclinical and clinical research on AAV-based CNS gene therapy and to discuss prospects for future development based on a new generation of vectors and delivery.
1 Lysogene, Neuilly sur Seine, France .
Michaël
M
Hocquemiller
1 Lysogene, Neuilly sur Seine, France .
Laura
L
Giersch
2 Université Paris Descartes , Paris, France .|3 INSERM UMR1169, Université Paris-Sud, Université Paris-Saclay , Orsay, France .|4 CEA, DSV, I2BM, MIRCen, Fontenay-aux-Roses, France .
Mickael
M
Audrain
1 Lysogene, Neuilly sur Seine, France .
Samantha
S
Parker
3 INSERM UMR1169, Université Paris-Sud, Université Paris-Saclay , Orsay, France .|4 CEA, DSV, I2BM, MIRCen, Fontenay-aux-Roses, France .
Nathalie
N
Cartier
D000818:Animals; D002493:Central Nervous System Diseases / Q000235:genetics / Q000628:therapy*; D000229:Dependovirus / Q000235:genetics*; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000008:administration & dosage*; D006801:Humans