Ontology highlight
ABSTRACT:
SUBMITTER: Meng J
PROVIDER: S-EPMC4728433 | biostudies-literature | 2016 Jan
REPOSITORIES: biostudies-literature
Meng Jinhong J Counsell John R JR Reza Mojgan M Laval Steven H SH Danos Olivier O Thrasher Adrian A Lochmüller Hanns H Muntoni Francesco F Morgan Jennifer E JE
Scientific reports 20160127
Autologous stem cells that have been genetically modified to express dystrophin are a possible means of treating Duchenne Muscular Dystrophy (DMD). To maximize the therapeutic effect, dystrophin construct needs to contain as many functional motifs as possible, within the packaging capacity of the viral vector. Existing dystrophin constructs used for transduction of muscle stem cells do not contain the nNOS binding site, an important functional motif within the dystrophin gene. In this proof-of-c ...[more]