… variable phenotype known as Becker muscular dystrophy or X-linked … by other genetic modifiers
distinct from the DMD gene, … binding protein 4 (LBP4) gene and osteopontin, encoded by …

… DUCHENNE MUSCULAR DYSTROPHY DMD is the most common form of muscular dystrophy
in childhood. Because the DMD gene (the gene encoding the protein dystrophin) is found …

… reported that dystrophin is a tumor suppressor gene and likely … that therapeutic approaches
developed for muscular dystrophies … modifiers, as well as setting up in vitro protocols of gene …

… Validation of genetic modifiers for Duchenne muscular dystrophy: a multicentre study
assessing SPP1 and LTBP4 variants. J Neurol Neurosurg Psychiatry. 2015;86:1060–5. …

… Gene therapy can refer to the introduction of a therapeutic nucleic acid (DNA, RNA, …
traditional gene therapy approaches of gene addition to supply a functional copy of the DMD gene, …

… The dystrophin gene is the largest gene described in human and its full length messenger
… and cardiac muscle, and also, with small amounts, in the brain. The DMD gene produces …

… muscular dystrophy (DMD) is an X-linked progressive muscle-wasting disorder that is caused
by a lack of functional dystrophin, a … for the structural integrity of muscle. As variants in the …

… We explore how genetic modifiers could be used to predict cardiorespiratory natural history
and how manipulation of such modifiers might represent a promising therapeutic strategy. …

… for the permanent correction of diverse genetic … of genetic mutations that cause muscular
dystrophies. Finally, we highlight current cell- and animal-based studies of muscular dystrophy …

… model of Lama2-mediated congenital muscular dystrophy, leading to significant
functional … -dCas9-mediated transcriptional modulation of modifier genes, ie, a mutation-independent …