New developments in gene editing for Duchenne muscular dystrophy

Nature Reviews Cardiology, Published online: 11 February 2020; doi:10.1038/s41569-020-0350-7A somatic gene editing therapy for the treatment of Duchenne muscular dystrophy (DMD) improves skeletal and cardiac muscle function and reduces cardiac arrhythmogenic vulnerability in a pig model of DMD and an in vitro model of human DMD.
Source: Nature Reviews Cardiology - Category: Cardiology Authors: Source Type: research

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(Natural News) A rare disease called limb-girdle muscular dystrophy D1 (LMGD1) makes even everyday actions such as climbing stairs, lifting objects and standing up from a chair difficult to do. Over time, it can even cause some people to lose the ability to walk. A recent study, however, shows that lithium can be a possible treatment for LMGD1. Researchers...
Source: - Category: Consumer Health News Source Type: news
ConclusionsPsychosocial interventions should address problems of anxiety and depression that people with MDs frequently experience, even through fostering parents ’ and childrens’ engagement coping over disengagement coping.
Source: Journal of Neurology - Category: Neurology Source Type: research
Publication date: Available online 26 March 2020Source: Meta GeneAuthor(s): Omid Daneshjoo, Ali hosseini, Masoud Garshasbi, Antonio Pizzuti
Source: Meta Gene - Category: Genetics & Stem Cells Source Type: research
The emergence of CRISPR-Cas9 gene-editing technologies and genome-wide CRISPR-Cas9 libraries enables efficient unbiased genetic screening that can accelerate the process of therapeutic discovery for genetic disorders. Here, we demonstrate the utility of a genome-wide CRISPR-Cas9 loss-of-function library to identify therapeutic targets for facioscapulohumeral muscular dystrophy (FSHD), a genetically complex type of muscular dystrophy for which there is currently no treatment. In FSHD, both genetic and epigenetic changes lead to misexpression of DUX4, the FSHD causal gene that encodes the highly cytotoxic DUX4 protein. We pe...
Source: Science Translational Medicine - Category: Biomedical Science Authors: Tags: Research Articles Source Type: research
Higher MMP2 abundance and gelatinolytic activity in 28- &70-d mdx compared with wild-type mice is alleviated in mdx mice with pre-natal taurine supplementation. Am J Physiol Cell Physiol. 2020 Mar 25;: Authors: Ren X, Xu H, Barker RG, Lamb GD, Murphy RM Abstract Duchenne muscular dystrophy (DMD) is a severe, progressive muscle wasting disorder that leads to early death. The mdx mouse is a naturally occurring mutant model for DMD. It lacks dystrophin and displays peak muscle cell necrosis at ~28 days (D28), but in contrast to DMD, mdx mice experience muscle regeneration by D70. We hypothesised that...
Source: American Journal of Physiology. Cell Physiology - Category: Cytology Authors: Tags: Am J Physiol Cell Physiol Source Type: research
Yokota Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disorder most commonly caused by mutations disrupting the reading frame of the dystrophin (DMD) gene. DMD codes for dystrophin, which is critical for maintaining the integrity of muscle cell membranes. Without dystrophin, muscle cells receive heightened mechanical stress, becoming more susceptible to damage. An active body of research continues to explore therapeutic treatments for DMD as well as to further our understanding of the disease. These efforts rely on having reliable animal models that accurately recapitulate disease pr...
Source: Genes - Category: Genetics & Stem Cells Authors: Tags: Review Source Type: research
AbstractObjectiveThe main aim was to explore the changes in hand-grip strength in patients with Duchenne muscular dystrophy (DMD) aged 5 –29 years. Secondary aims were to test the effect of mutation, ambulatory status and glucocorticoid use on grip strength and its changes over time and to compute the number of subjects needed for a clinical trial to stabilize grip strength.MethodsThe analysis was performed on data collected during five international natural history studies on a cohort of DMD patients. Two hundred and two patients with genetically proven DMD were pooled from five different natural history studie...
Source: Journal of Neurology - Category: Neurology Source Type: research
Publication date: April 2020Source: Molecular and Cellular Neuroscience, Volume 104Author(s): Riccardo Bianchi, Wouter Eilers, Federica Pellati, Lorenzo Corsi, Helen Foster, Keith Foster, Francesco Tamagnini
Source: Molecular and Cellular Neuroscience - Category: Neuroscience Source Type: research
A medicine developed by EU-funded researchers has been approved to treat children with the degenerative and fatal genetic disease Duchenne muscular dystrophy. A major clinical trial is expected to announce positive results soon.
Source: EUROPA - Research Information Centre - Category: Research Source Type: news
Pelliccia Common fragile sites (CFSs) are particularly vulnerable regions of the genome that become visible as breaks, gaps, or constrictions on metaphase chromosomes when cells are under replicative stress. Impairment in DNA replication, late replication timing, enrichment of A/T nucleotides that tend to form secondary structures, the paucity of active or inducible replication origins, the generation of R-loops, and the collision between replication and transcription machineries on particularly long genes are some of the reported characteristics of CFSs that may contribute to their tissue-specific fragility. Here, we ...
Source: Genes - Category: Genetics & Stem Cells Authors: Tags: Article Source Type: research
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