Recent progress and considerations for AAV gene therapies targeting the central nervous system.

ABSTRACT:

Background

Neurodevelopmental disorders, as a class of diseases, have been particularly difficult to treat even when the underlying cause(s), such as genetic alterations, are understood. What treatments do exist are generally not curative and instead seek to improve quality of life for affected individuals. The advent of gene therapy via gene replacement offers the potential for transformative therapies to slow or even stop disease progression for current patients and perhaps minimize or prevent the appearance of symptoms in future patients.

Main body

This review focuses on adeno-associated virus (AAV) gene therapies for diseases of the central nervous system. An overview of advances in AAV vector design for therapy is provided, along with a description of current strategies

SUBMITTER: Lykken EA 

PROVIDER: S-EPMC5960126 | biostudies-literature | 2018 May

REPOSITORIES: biostudies-literature