Article
Efficient and specific repair of sickle beta-globin RNA by trans-splicing ribozymes.
RNA (New York, N.Y.) - 1 Oct 2003
Byun Jonghoe, Lan Ning, Long Meredith, Sullenger Bruce A
Abstract excerpt
Previously we demonstrated that a group I ribozyme can perform trans-splicing to repair sickle beta-globin transcripts upon transfection of in vitro transcribed ribozyme into mammalian cells. Here, we sought to develop expression cassettes that would yield high levels of active ribozyme after gene transfer. Our initial expression constructs were designed to generate trans-slicing ribozymes identical to those used...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.