Current status of gene therapy for muscle diseases.
The progress made in vector technology and preclinical evaluation of molecular and gene therapy for inherited muscle diseases led to initiation of the first clinical trials using plasmid and adeno-associated virus vectors, as well as antisense oligonucleotides. The scope of this review is to discuss the current status of gene therapy for muscular dystrophy and to highlight recent advances in viral, cell-based as well as molecular therapies.
Thirion
Christian
C
Laboratory of Molecular Myology, Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munchen, Germany. Hanns.Lochmueller@med.uni-muenchen.de
Lochmüller
Hanns
H
D000818:Animals; D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D009136:Muscular Dystrophies / Q000235:genetics / Q000628:therapy*; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use; D033581:Stem Cell Transplantation / Q000379:methods; D014780:Viruses / Q000235:genetics
D016376:Oligonucleotides, Antisense