Article
In vivo assessment of mutations in OTC for dominant-negative effects following rAAV2/8-mediated gene delivery to the mouse liver.
Gene therapy - 1 Jun 2009
Ginn S L, Cunningham S C, Zheng M, Spinoulas A, Carpenter K H, Alexander I E
Abstract excerpt
Mutant proteins have the potential to exert dominant-negative effects that might limit the therapeutic efficacy of their wild-type counterparts after gene transfer. For ornithine transcarbamylase (OTC) deficiency, in vitro studies have suggested the presence of dominant-negative effects, however, supporting in vivo studies have not been conducted. In this study, we exploited the capacity of recombinant...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.