Ontology highlight
ABSTRACT:
SUBMITTER: Choudhury SR
PROVIDER: S-EPMC5088762 | biostudies-literature | 2016 Aug
REPOSITORIES: biostudies-literature
Choudhury Sourav R SR Fitzpatrick Zachary Z Harris Anne F AF Maitland Stacy A SA Ferreira Jennifer S JS Zhang Yuanfan Y Ma Shan S Sharma Rohit B RB Gray-Edwards Heather L HL Johnson Jacob A JA Johnson Aime K AK Alonso Laura C LC Punzo Claudio C Wagner Kathryn R KR Maguire Casey A CA Kotin Robert M RM Martin Douglas R DR Sena-Esteves Miguel M
Molecular therapy : the journal of the American Society of Gene Therapy 20160427 7
Adeno-associated viral (AAV) vectors have shown promise as a platform for gene therapy of neurological disorders. Achieving global gene delivery to the central nervous system (CNS) is key for development of effective therapies for many of these diseases. Here we report the isolation of a novel CNS tropic AAV capsid, AAV-B1, after a single round of in vivo selection from an AAV capsid library. Systemic injection of AAV-B1 vector in adult mice and cat resulted in widespread gene transfer throughou ...[more]