NEW FINDINGS: What is the topic of this review? This review highlights recent progress in genetically based therapies targeting the primary defect of @entity1555 . What advances does it highlight? Over the last two decades, considerable progress has been made in understanding the mechanisms underlying @entity1555 , leading to the development of genetic therapies. These include manipulation of the expression of the gene or related genes, the splicing of the gene and its translation, and replacement of the gene using viral approaches. @entity1555 is a lethal @entity1112 caused by mutations in the @entity4714 gene. In the absence of the @entity4714 protein, the link between the cytoskeleton and extracellular matrix is destroyed, and this severely compromises the strength, flexibility and stability of muscle fibres. The devastating consequence is progressive @entity2057 and @entity204 in @entity1555 @entity1 . There is currently no cure, and despite exhaustive palliative care, @entity1 are restricted to a wheelchair by the age of 12  years and usually succumb to @entity299 in their late 20s. This review provides an update on the current genetically based therapies and clinical trials that target or compensate for the primary defect of this disease. These include @entity4714 gene-replacement strategies, genetic modification techniques to restore @entity4714 expression, and modulation of the @entity4714 homologue, @entity28243 , as a surrogate to re-establish muscle function.

Advances in genetic therapeutic strategies for XXXX .

@entity1 :: ('9606', 'Species') :: ['patients'] @entity1112 :: ('MESH:D040181', 'Disease') :: ['X-linked disorder'] @entity28243 :: ('7402', 'Gene') :: ['utrophin'] @entity1555 :: ('MESH:D020388', 'Disease') :: ['Duchenne muscular dystrophy'] @entity4714 :: ('1756', 'Gene') :: ['dystrophin'] @entity299 :: ('MESH:D006331', 'Disease') :: ['cardiac or respiratory complications'] @entity2057 :: ('MESH:D009133', 'Disease') :: ['muscle wasting'] @entity204 :: ('MESH:D003643', 'Disease') :: ['premature death']
@entity1555 :: (MESH:D020388,Disease) :: ['Duchenne muscular dystrophy']
