A Boost for Muscle with Gene Therapy
Jeffrey S. Chamberlain
Abstract
Gene therapy for disorders affecting muscle, such as Duchenne’s muscular dystrophy and myotubular myopathy, has been challenging, in large part owing to the difficulty of delivering the therapeutic gene to cells within the mass of muscle tissue. A recent study of the adeno-associated virus capsid provides a candidate vector for targeting muscle cells.
Topics & Concepts
Genetic enhancementMedicineDuchenne muscular dystrophyAdeno-associated virusMuscular dystrophyMuscle disorderVector (molecular biology)MyopathyGeneMyocyteMuscle diseaseSkeletal muscleViral vectormdx mouseBioinformaticsDystrophinPathologyGeneticsAnatomyDiseaseInternal medicineBiologyRecombinant DNAVirus-based gene therapy researchViral Infectious Diseases and Gene Expression in InsectsCAR-T cell therapy research