Article
Development of a Single Vector System that Enhances Trans-Splicing of SMN2 Transcripts
21 Oct 2008
Abstract excerpt
RNA modalities are developing as a powerful means to re-direct pathogenic pre-mRNA splicing events. Improving the efficiency of these molecules in vivo is critical as they move towards clinical applications. Spinal muscular atrophy (SMA) is caused by loss of SMN1. A nearly identical copy gene called SMN2 produces low levels of functional protein due to alternative splicing. We previously reported a trans-splicing...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
