TY - JOUR
T1 - Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies
AU - Selvaraj, Sridhar
AU - Kyba, Michael
AU - Perlingeiro, Rita C.R.
N1 - Publisher Copyright:
© 2019 Elsevier Ltd
PY - 2019/9
Y1 - 2019/9
N2 - 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.
AB - 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.
KW - cell therapy
KW - gene correction
KW - muscular dystrophy
KW - myogenic progenitors
KW - pluripotent stem cells
UR - http://www.scopus.com/inward/record.url?scp=85071775762&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85071775762&partnerID=8YFLogxK
U2 - 10.1016/j.molmed.2019.07.004
DO - 10.1016/j.molmed.2019.07.004
M3 - Review article
C2 - 31473142
AN - SCOPUS:85071775762
SN - 1471-4914
VL - 25
SP - 803
EP - 816
JO - Trends in Molecular Medicine
JF - Trends in Molecular Medicine
IS - 9
ER -