Article
AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Nov 2004
Yoshimura Madoka, Sakamoto Miki, Ikemoto Madoka, Mochizuki Yasushi, Yuasa Katsutoshi, Miyagoe-Suzuki Yuko, Takeda Shin'ichi
Abstract excerpt
Duchenne muscular dystrophy (DMD) is a lethal disorder of skeletal muscle caused by mutations in the dystrophin gene. Adeno-associated virus (AAV) vector-mediated gene therapy is a promising approach to the disease. Although a rod-truncated microdystrophin gene has been proven to ameliorate dystrophic phenotypes, the level of microdystrophin expression required for effective gene therapy by an AAV vector has not...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.