Ontology highlight
ABSTRACT:
SUBMITTER: Chadderton N
PROVIDER: S-EPMC2835099 | biostudies-literature | 2009 Apr
REPOSITORIES: biostudies-literature
Chadderton Naomi N Millington-Ward Sophia S Palfi Arpad A O'Reilly Mary M Tuohy Gearóid G Humphries Marian M MM Li Tiansen T Humphries Peter P Kenna Paul F PF Farrar G Jane GJ
Molecular therapy : the journal of the American Society of Gene Therapy 20090127 4
Mutational heterogeneity represents one of the greatest barriers impeding the progress toward the clinic of gene therapies for many dominantly inherited disorders. A general strategy of gene suppression in conjunction with replacement has been proposed to overcome this mutational heterogeneity. In the current study, various aspects of this strategy are explored for a dominant form of the retinal degeneration, retinitis pigmentosa (RP), caused by mutations in the rhodopsin gene (RHO-adRP). While ...[more]