Viral vectors and delivery strategies for CNS gene therapy

This review aims to provide a broad overview of the targets, challenges and potential for gene therapy in the CNS, citing specific examples. There are a broad range of therapeutic targets, with very different requirements for a suitable viral vector. By utilizing different vector tropisms, novel rou...

Full description

Saved in:
Bibliographic Details
Published inTherapeutic delivery Vol. 1; no. 4; p. 517
Main Authors Gray, Steven J, Woodard, Kenton T, Samulski, R Jude
Format Journal Article
LanguageEnglish
Published England 01.10.2010
Subjects
Animals
Central Nervous System Diseases - genetics
Central Nervous System Diseases - therapy
Gene Transfer Techniques
Genetic Therapy - methods
Genetic Vectors
Humans
Viruses - genetics
Online AccessGet more information

Cover

More Information
Summary:This review aims to provide a broad overview of the targets, challenges and potential for gene therapy in the CNS, citing specific examples. There are a broad range of therapeutic targets, with very different requirements for a suitable viral vector. By utilizing different vector tropisms, novel routes of administration and engineered promoter control, transgenes can be targeted to specific therapeutic applications. Viral vectors have proven efficacious in preclinical models for several disease applications, spurring several clinical trials. While the field has pushed the limits of existing adeno-associated virus-based vectors, a next generation of vectors based on rational engineering of viral capsids should expand the application of gene therapy to be more effective in specific therapeutic applications.
ISSN:2041-5990
DOI:10.4155/tde.10.50