Year: 2014
Type: article
Abstract: Rescue of information from mutated genes, as opposed to replacing them, is a particularly promising approach to therapy for Duchenne muscular dystrophy (DMD). Because this disease commonly arises from relatively small mutations in the very large dystrophin gene, most of the structural information is often left intact and available for use if the interfering effect of the mutated region could be removed from the gene transcript. Practical application of this idea in DMD patients has been moved an important step forward in the recent publication by Le Guiner et al.
Source: Molecular Therapy
DOI: 10.1038/mt.2014.189
Author: Terence Partridge
Institution: Children's National
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $4,500
OpenAlex ID: W2047005331