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Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies.

ABSTRACT: Pluripotent stem cells (PSCs) represent an attractive cell source for treating muscular dystrophies (MDs) since they easily allow for the generation of large numbers of highly regenerative myogenic progenitors. Using reprogramming technology, patient-specific PSCs have been derived for several types of MDs, and genome editing has allowed correction of mutations, opening the opportunity for their therapeutic application in an autologous transplantation setting. However, there has been limited progress on preclinical studies that validate the therapeutic potential of these gene corrected PSC-derived myogenic progenitors. In this review, we highlight the major research advances, challenges, and future prospects towards the development of PSC-based therapeutics for MDs.

SUBMITTER: Selvaraj S

PROVIDER: S-EPMC6721995 | biostudies-literature | 2019 Sep

REPOSITORIES: biostudies-literature

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Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies.

Selvaraj Sridhar S Kyba Michael M Perlingeiro Rita C R RCR

Trends in molecular medicine 20190901 9

Pluripotent stem cells (PSCs) represent an attractive cell source for treating muscular dystrophies (MDs) since they easily allow for the generation of large numbers of highly regenerative myogenic progenitors. Using reprogramming technology, patient-specific PSCs have been derived for several types of MDs, and genome editing has allowed correction of mutations, opening the opportunity for their therapeutic application in an autologous transplantation setting. However, there has been limited pro ...[more]

PMID: 31473142

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