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Thérapie génique du système nerveux central: Considérations générales sur les vecteurs viraux pour le transfert de gène dans le cerveau

Ché Serguera 1 Alexis-Pierre Bemelmans 1 
1 LMN - Laboratoire des Maladies Neurodégénératives - UMR 9199
MIRCEN - Service MIRCEN : DRF/JACOB, CNRS - Centre National de la Recherche Scientifique : UMR 9199
Abstract : The last decade have nourished strong doubts on the beneficial prospects of gene therapy for curing fatal diseases. However, this climate of reservations is currently being transcended by the publication of several successful clinical protocols, restoring confidence in the opportunity of therapeutic gene transfer. A strong sign of this present enthusiasm for gene therapy by clinicians and industrials is the market approval of the therapeutic viral vector Glybera, the first commercial product in Europe of this class of drug. This new field of medicine is particularly attractive when considering therapies for a number of neurological disorders, most of which are desperately waiting for a satisfactory treatment. The central nervous system is indeed a very compliant organ where gene transfer can be stable and successful if provided through an appropriate strategy. The purpose of this review is to present the characteristics of the most efficient virus-derived vectors used by researchers and clinicians to genetically-modify particular cell types or whole regions of the brain. In addition, we discuss major issues regarding side effects such as genotoxicity and immune response associated to the use of these vectors
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Submitted on : Monday, April 25, 2022 - 10:58:25 AM
Last modification on : Wednesday, April 27, 2022 - 3:17:04 AM

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Ché Serguera, Alexis-Pierre Bemelmans. Thérapie génique du système nerveux central: Considérations générales sur les vecteurs viraux pour le transfert de gène dans le cerveau. Revue Neurologique, Elsevier Masson, 2014, 170 (12), pp.727-738. ⟨10.1016/j.neurol.2014.09.004⟩. ⟨cea-03650651⟩

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