1. Cystic Fibrosis
(NIH) Doctors and scientists are now looking toward gene therapy, specifically SmaRT gene therapy as a possible cure. ... (Thompson) Conventional gene therapy involves the addition of a corrected gene into the patients DNA. (Thomson) SmaRT (spliceosome-mediated RNA trans-splicing) therapy works a little differently. ... Do we understand the pathology of the disease enough to use this therapy? ... CF may be the first disease controlled or cured with genetic therapy. ...
- Word Count: 1147
- Approx Pages: 5
- Has Bibliography
- Grade Level: Undergraduate