Article

The last decade has nourished strong doubts on the beneficial prospects of gene
therapy for curing fatal diseases. However, this climate of reservation is
currently being transcended by the publication of several successful clinical
protocols, restoring confidence in the appropriateness 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.
CI - Copyright (c) 2014 Elsevier Masson SAS. All rights reserved.

Langue : ANGLAIS