Ontology highlight
ABSTRACT:
SUBMITTER: Rodino-Klapac LR
PROVIDER: S-EPMC2839222 | biostudies-literature | 2010 Jan
REPOSITORIES: biostudies-literature
Rodino-Klapac Louise R LR Montgomery Chrystal L CL Bremer William G WG Shontz Kimberly M KM Malik Vinod V Davis Nancy N Sprinkle Spencer S Campbell Katherine J KJ Sahenk Zarife Z Clark K Reed KR Walker Christopher M CM Mendell Jerry R JR Chicoine Louis G LG
Molecular therapy : the journal of the American Society of Gene Therapy 20091110 1
Animal models for Duchenne muscular dystrophy (DMD) have species limitations related to assessing function, immune response, and distribution of micro- or mini-dystrophins. Nonhuman primates (NHPs) provide the ideal model to optimize vector delivery across a vascular barrier and provide accurate dose estimates for widespread transduction. To address vascular delivery and dosing in rhesus macaques, we have generated a fusion construct that encodes an eight amino-acid FLAG epitope at the C-terminu ...[more]