A Perspective on the Potential of Human iPS Cell-Based Therapies for Muscular Dystrophies: Advancements so far and Hurdles to Overcome

por Darabi, Radbod, Perlingeiro, Rita C.R.
Publicado 2013

Transplantation studies reveal internuclear transfer of toxic RNA in engrafted muscles of myotonic dystrophy 1 mice

por Mondragon-Gonzalez, Ricardo, Azzag, Karim, Selvaraj, Sridhar, Yamamoto, Ami, Perlingeiro, Rita C.R.
Publicado 2019

Genomic Safe Harbor Expression of PAX7 for the Generation of Engraftable Myogenic Progenitors

por Kim, Hyunkee, Selvaraj, Sridhar, Kiley, James, Azzag, Karim, Garay, Bayardo I., Perlingeiro, Rita C.R.
Publicado 2020

Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors

por Kim, Jaemin, Magli, Alessandro, Chan, Sunny S.K., Oliveira, Vanessa K.P., Wu, Jianbo, Darabi, Radbod, Kyba, Michael, Perlingeiro, Rita C.R.
Publicado 2017

In vitro expanded skeletal myogenic progenitors from pluripotent stem cell-derived teratomas have high engraftment capacity

por Xie, Ning, Chu, Sabrina N., Azzag, Karim, Schultz, Cassandra B., Peifer, Lindsay N., Kyba, Michael, Perlingeiro, Rita C.R., Chan, Sunny S.K.
Publicado 2021

Defective autophagy and increased apoptosis contribute toward the pathogenesis of FKRP-associated muscular dystrophies

por Ortiz-Cordero, Carolina, Bincoletto, Claudia, Dhoke, Neha R., Selvaraj, Sridhar, Magli, Alessandro, Zhou, Haowen, Kim, Do-Hyung, Bang, Anne G., Perlingeiro, Rita C.R.
Publicado 2021

NAD+ enhances ribitol and ribose rescue of α-dystroglycan functional glycosylation in human FKRP-mutant myotubes

por Ortiz-Cordero, Carolina, Magli, Alessandro, Dhoke, Neha R, Kuebler, Taylor, Selvaraj, Sridhar, Oliveira, Nelio AJ, Zhou, Haowen, Sham, Yuk Y, Bang, Anne G, Perlingeiro, Rita CR
Publicado 2021

Screening identifies small molecules that enhance the maturation of human pluripotent stem cell-derived myotubes

por Selvaraj, Sridhar, Mondragon-Gonzalez, Ricardo, Xu, Bin, Magli, Alessandro, Kim, Hyunkee, Lainé, Jeanne, Kiley, James, Mckee, Holly, Rinaldi, Fabrizio, Aho, Joy, Tabti, Nacira, Shen, Wei, Perlingeiro, Rita CR
Publicado 2019

An ex vivo Gene Therapy Approach to Treat Muscular Dystrophy Using inducible Pluripotent Stem Cells

por Filareto, Antonio, Parker, Sarah, Darabi, Radbod, Borges, Luciene, Iacovino, Michelina, Schaaf, Tory, Mayerhofer, Timothy, Chamberlain, Jeffrey S, Ervasti, James M., McIvor, R. Scott, Kyba, Michael, Perlingeiro, Rita C.R.
Publicado 2013

Pax3-induced expansion enables the genetic correction of dystrophic satellite cells

por Filareto, Antonio, Rinaldi, Fabrizio, Arpke, Robert W., Darabi, Radbod, Belanto, Joseph J., Toso, Erik A., Miller, Auston Z., Ervasti, James M., McIvor, R. Scott, Kyba, Michael, Perlingeiro, Rita CR
Publicado 2015

A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy

por Dhoke, Neha R., Kim, Hyunkee, Selvaraj, Sridhar, Azzag, Karim, Zhou, Haowen, Oliveira, Nelio A.J., Tungtur, Sudheer, Ortiz-Cordero, Carolina, Kiley, James, Lu, Qi Long, Bang, Anne G., Perlingeiro, Rita C.R.
Publicado 2021