Systemic cell therapy for muscular dystrophies
نویسندگان
چکیده
منابع مشابه
Advances in gene therapy for muscular dystrophies
Duchenne muscular dystrophy (DMD) is a recessive lethal inherited muscular dystrophy caused by mutations in the gene encoding dystrophin, a protein required for muscle fibre integrity. So far, many approaches have been tested from the traditional gene addition to newer advanced approaches based on manipulation of the cellular machinery either at the gene transcription, mRNA processing or transl...
متن کاملThe Pathogenesis and Therapy of Muscular Dystrophies.
Current molecular genomic approaches to human genetic disorders have led to an explosion in the identification of the genes and their encoded proteins responsible for these disorders. The identification of the gene altered by mutations in Duchenne and Becker muscular dystrophy was one of the earliest examples of this paradigm. The nearly 30 years of research partly outlined here exemplifies the...
متن کاملMuscular dystrophies, the cytoskeleton and cell adhesion.
Muscular dystrophies are associated with mutations in genes encoding several classes of proteins. These range from extracellular matrix and integral membrane proteins to cytoskeletal proteins, but also include a heterogeneous group of proteins including proteases, nuclear proteins, and signalling molecules. Muscular dystrophy phenotypes have also become evident in studies on various knockout mi...
متن کاملStem Cell Based Therapy for Muscular Dystrophies: Cell Types and Environmental Factors Influencing Their Efficacy
Muscular dystrophies are inherited disorders in which muscle fibers are unusually susceptible to damage, leading to progressive loss of muscle structure and function. Some types of muscular dystrophy affect heart muscles, other involuntary muscles and other organs. The most common form of muscular dystrophy, Duchenne Muscular Dystrophy (DMD), is due to genetic deficiency of the protein dystroph...
متن کاملEnhancing Myoblast Fusion for Therapy of Muscular Dystrophies
Skeletal muscle is a major organ comprising 30-40% of the human body mass. The coordination of processes resulting in mature muscle requires many genes, and their loss can result in debilitating muscle disorders. Of the strategies being developed to cure muscle diseases, enhancement of the natural process of muscle cell fusion in existing or introduced myogenic cells has great therapeutic poten...
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ژورنال
عنوان ژورنال: Stem Cell Reviews and Reports
سال: 2020
ISSN: 2629-3269,2629-3277
DOI: 10.1007/s12015-020-10100-y