Recent gene therapy advancements for neurological diseases.

[krabbe disease]

The past few years have seen rapid advancements in vector-mediated gene transfer to the nervous system and modest successes in human gene therapy trials . The purpose of this review is to describe commonly-used viral gene transfer vectors and recent advancements towards producing meaningful gene -based treatments for central nervous system (CNS ) disorders . Gene therapy trials for Canavan disease , Batten disease , adrenoleukodystrophy , and Parkinson 's disease are discussed to illustrate the current state of clinical gene transfer to the CNS . Preclinical studies are under way for a number of diseases , primarily lysosomal storage disorders , using a newer generation of vectors and delivery strategies . Relevant studies in animal models are highlighted for Mucopolysaccharidosis IIIB and Krabbe disease to provide a prelude for what can be expected in the coming years for human gene transfer trials , using recent advancements in gene transfer technology . In conclusion , recent improvements in CNS gene transfer technology are expected to significantly increase the degree of disease rescue in future CNS-directed clinical trials , exceeding the modest clinical successes that have been observed so far .