Enhanced selective gene delivery to neural stem cells in vivo by an adeno-associated viral variant.

ABSTRACT: Neural stem cells (NSCs) are defined by their ability to self-renew and to differentiate into mature neuronal and glial cell types. NSCs are the subject of intense investigation, owing to their crucial roles in neural development and adult brain function and because they present potential targets for gene and cell replacement therapies following injury or disease. Approaches to specifically genetically perturb or modulate NSC function would be valuable for either motivation. Unfortunately, most gene delivery vectors are incapable of efficient or specific gene delivery to NSCs in vivo. Vectors based on adeno-associated virus (AAV) present a number of advantages and have proven increasingly successful in clinical trials. However, natural AAV variants are inefficient in transducing NSCs. We p

SUBMITTER: Kotterman MA 

PROVIDER: S-EPMC4440921 | biostudies-literature | 2015 May

REPOSITORIES: biostudies-literature