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

Generation of human myogenic progenitors from pluripotent stem cells for in vivo regeneration

por Kim, Hyunkee, Perlingeiro, Rita C. R.
Publicado 2022

Recapitulating muscle disease phenotypes with myotonic dystrophy 1 induced pluripotent stem cells: a tool for disease modeling and drug discovery

por Mondragon-Gonzalez, Ricardo, Perlingeiro, Rita C. R.
Publicado 2018

Development of allogeneic iPS cell‐based therapy: from bench to bedside

por McKenna, David H, Perlingeiro, Rita C R
Publicado 2022

Treatment with rGDF11 does not improve the dystrophic muscle pathology of mdx mice

por Rinaldi, Fabrizio, Zhang, Yu, Mondragon-Gonzalez, Ricardo, Harvey, Jeffrey, Perlingeiro, Rita C. R.
Publicado 2016

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

Stimulation of Cardiomyocyte Proliferation Is Dependent on Species and Level of Maturation

por Yücel, Dogacan, Garay, Bayardo I., Perlingeiro, Rita C. R., van Berlo, Jop H.
Publicado 2022

Pluripotent stem cell-derived skeletal muscle fibers preferentially express myosin heavy-chain isoforms associated with slow and oxidative muscles

por Incitti, Tania, Magli, Alessandro, Jenkins, Asher, Lin, Karena, Yamamoto, Ami, Perlingeiro, Rita C. R.
Publicado 2020

Transplantation of PSC-derived myogenic progenitors counteracts disease phenotypes in FSHD mice

por Azzag, Karim, Bosnakovski, Darko, Tungtur, Sudheer, Salama, Peter, Kyba, Michael, Perlingeiro, Rita C. R.
Publicado 2022

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

Chromatin accessibility profiling identifies evolutionary conserved loci in activated human satellite cells

por Chow, Lisa S., Bosnakovski, Darko, Mashek, Douglas G., Kyba, Michael, Perlingeiro, Rita C. R., Magli, Alessandro
Publicado 2021

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

Muscle progenitor specification and myogenic differentiation are associated with changes in chromatin topology

por Zhang, Nan, Mendieta-Esteban, Julen, Magli, Alessandro, Lilja, Karin C., Perlingeiro, Rita C. R., Marti-Renom, Marc A., Tsirigos, Aristotelis, Dynlacht, Brian David
Publicado 2020

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

Pax3 cooperates with Ldb1 to direct local chromosome architecture during myogenic lineage specification

por Magli, Alessandro, Baik, June, Pota, Pruthvi, Cordero, Carolina Ortiz, Kwak, Il-Youp, Garry, Daniel J., Love, Paul E., Dynlacht, Brian D., Perlingeiro, Rita C. R.
Publicado 2019

Efficient engraftment of pluripotent stem cell-derived myogenic progenitors in a novel immunodeficient mouse model of limb girdle muscular dystrophy 2I

por Azzag, Karim, Ortiz-Cordero, Carolina, Oliveira, Nelio A. J., Magli, Alessandro, Selvaraj, Sridhar, Tungtur, Sudheer, Upchurch, Weston, Iaizzo, Paul A., Lu, Qi Long, Perlingeiro, Rita C. R.
Publicado 2020

Endoglin integrates BMP and Wnt signalling to induce haematopoiesis through JDP2

por Baik, June, Magli, Alessandro, Tahara, Naoyuki, Swanson, Scott A., Koyano-Nakagawa, Naoko, Borges, Luciene, Stewart, Ron, Garry, Daniel J., Kawakami, Yasuhiko, Thomson, James A., Perlingeiro, Rita C. R.
Publicado 2016

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

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