@entity1555 ( @entity1555 ) is a @entity74 affecting about one in every 3,500 @entity1 . This X-linked pathology is due to the absence of @entity4714 in muscle fibers. This lack of @entity4714 leads to the progressive @entity121 that is often responsible for the death of the @entity1555 @entity1 during the third decade of their life. There are currently no curative treatments for this disease but different therapeutic approaches are being studied. Gene therapy consists of introducing a transgene coding for full-length or a truncated version of @entity4714 complementary DNA (cDNA) in muscles, whereas pharmaceutical therapy includes the use of chemical/biochemical substances to restore @entity4714 expression or alleviate the @entity1555 phenotype. Over the past years, many potential drugs were explored. This led to several clinical trials for @entity801 and ataluren ( @entity14045 ) allowing stop codon read-through. An alternative approach is to induce the expression of an internally deleted, partially functional @entity4714 protein through exon skipping. The vectors and the methods used in gene therapy have been continually improving in order to obtain greater encapsidation capacity and better transduction efficiency. The most promising experimental approaches using pharmaceutical and gene therapies are reviewed in this article.

Current status of pharmaceutical and genetic therapeutic approaches to treat XXXX .

@entity1 :: ('9606', 'Species') :: ['patients', 'boys'] @entity801 :: ('MESH:D005839', 'Chemical') :: ['gentamicin'] @entity14045 :: ('MESH:C515878', 'Chemical') :: ['PTC124'] @entity1555 :: ('MESH:D020388', 'Disease') :: ['Duchenne muscular dystrophy', 'DMD'] @entity4714 :: ('1756', 'Gene') :: ['dystrophin'] @entity74 :: ('MESH:D030342', 'Disease') :: ['genetic disease'] @entity121 :: ('MESH:D009135', 'Disease') :: ['muscle degeneration']
@entity1555 :: (MESH:D020388,Disease) :: ['DMD']
