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Eur J Pharmacol ; 912: 174568, 2021 Dec 05.
Artigo em Inglês | MEDLINE | ID: mdl-34656607

RESUMO

Muscular dystrophy is a well-known genetically heterogeneous group of rare muscle disorders. This progressive disease causes the breakdown of skeletal muscles over time and leads to grave weakness. This breakdown is caused by a diverse pattern of mutations in dystrophin and dystrophin associated protein complex. These mutations lead to the production of altered proteins in response to which, the body stimulates production of various cytokines and immune cells, particularly reactive oxygen species and NFκB. Immune cells display/exhibit a dual role by inducing muscle damage and muscle repair. Various anti-oxidants, anti-inflammatory and glucocorticoid drugs serve as potent therapeutics for muscular dystrophy. Along with the above mentioned therapeutics, induced pluripotent stem cells also serve as a novel approach paving a way for personalized treatment. These pluripotent stem cells allow regeneration of large numbers of regenerative myogenic progenitors that can be administered in muscular dystrophy patients which assist in the recovery of lost muscle fibers. In this review, we have summarized gene-editing, immunological and induced pluripotent stem cell based therapeutics for muscular dystrophy treatment.


Assuntos
Edição de Genes/métodos , Células-Tronco Pluripotentes Induzidas , Distrofias Musculares/genética , Distrofias Musculares/imunologia , Animais , Distrofina/genética , Humanos , Distrofias Musculares/metabolismo , Distrofias Musculares/terapia , NF-kappa B/antagonistas & inibidores , NF-kappa B/metabolismo , Espécies Reativas de Oxigênio/antagonistas & inibidores , Espécies Reativas de Oxigênio/metabolismo
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