Myoblast transplantation: the current status of a potential therapeutic tool for myopathies.

作者: Daniel Skuk , Jacques P. Tremblay

DOI: 10.1023/A:1025425823322

关键词: BioinformaticsImmunologyDistribution (pharmacology)ToxicityInduced pluripotent stem cellBiologyMyocyteTransplantationImmunosuppressionIn patientDuchenne muscular dystrophy

摘要: The implantation of cultured myogenic cells into the body (myoblast transplantation) is an experimental strategy that being explored for potential treatment myopathies. Its benefits should be: (1) to slow down or stop muscle degeneration, and/or (2) increase force in wasted muscles. For these objectives, myoblast transplantation may act by two actions: genetic complementation (as a vehicle normal genes case myopathies), and increasing pool muscle. During last decade, seemed stagnant contradiction experiments producing good results mice, against poor human trials. This was apparent, since conditions used mouse models were largely different from those dystrophic patients. Our monkey demonstrated promising can be observed large muscles primates, but under differ previously These are: appropriate immunosuppression, careful distribution sufficient quantities myoblasts recipient Most work on addressed improve this method by: reducing avoiding toxicity sustained favoring donor-myoblast migration muscle, (3) defining factors implicated early donor-cell survival following intramuscular implantation. Other research subjects field are use pluripotent stem instead satellite cells, delivery exogenous blood stream.

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