Journal
HUMAN GENE THERAPY
Volume 28, Issue 3, Pages 228-230Publisher
MARY ANN LIEBERT, INC
DOI: 10.1089/hum.2017.037
Keywords
eye; intravenous; single-stranded; CNS
Categories
Funding
- Massachusetts Lions Eye Research Fund
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In order to pursue a clinical gene therapy for a human neurologic disease, it is often necessary to perform proof-of-concept trials in mouse models of that disease. In order to demonstrate a potential clinical efficacy, one must be able to select an appropriate vector and route of delivery for the appropriate age group in the disease model. Since many diseases require correction early in life, investigators often need to deliver recombinant adeno-associated viral (rAAV) vectors to neonatal mice. Herein, general central nervous system expression patterns of nuclear GFP following delivery of rAAV by three different routes are reported.
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