Autologous cell therapy approach for Duchenne muscular dystrophy using PiggyBac transposons and mesoangioblasts
… Duchenne muscular dystrophy (DMD) is a lethal muscle-wasting disease currently
without cure. We investigated the use of the PiggyBac transposon for full-length dystrophin …
without cure. We investigated the use of the PiggyBac transposon for full-length dystrophin …
Therapeutic strategies for Duchenne muscular dystrophy: an update
… type of muscular dystrophy, Duchenne muscular dystrophy (DMD), is caused by mutations in
the X-linked dystrophin gene… Clinical data demonstrated that muscles treated with myoblast …
the X-linked dystrophin gene… Clinical data demonstrated that muscles treated with myoblast …
Creation of dystrophin expressing chimeric cells of myoblast origin as a novel stem cell based therapy for Duchenne muscular dystrophy
M Siemionow, J Cwykiel, A Heydemann… - Stem Cell Reviews and …, 2018 - Springer
… Over the past decade different stem cell (SC) based approaches were tested to treat Duchenne
Muscular Dystrophy (DMD), a lethal X-linked disorder caused by mutations in dystrophin …
Muscular Dystrophy (DMD), a lethal X-linked disorder caused by mutations in dystrophin …
Transplantation of dystrophin expressing chimeric human cells of myoblast/mesenchymal stem cell origin improves function in Duchenne muscular dystrophy model
M Siemionow, E Szilagyi, J Cwykiel… - Stem cells and …, 2021 - liebertpub.com
… dystrophin expression correlating with improved function after transplantation of dystrophin
expressing chimeric (DEC) cells of myoblast … cell line of myoblasts and mesenchymal stem …
expressing chimeric (DEC) cells of myoblast … cell line of myoblasts and mesenchymal stem …
Empowering muscle stem cells for the treatment of duchenne muscular dystrophy
RL Filippelli, NC Chang - Cells Tissues Organs, 2022 - karger.com
… effects on muscle health and is an underlying cause of muscle … cell dysfunction in Duchenne
muscular dystrophy (DMD), to … are transferred to DMD muscle tissue during myoblast fusion, …
muscular dystrophy (DMD), to … are transferred to DMD muscle tissue during myoblast fusion, …
Stem cell-based therapies for Duchenne muscular dystrophy
… cell transplantation and numerous efforts to optimize cell culture conditions in a lab setting,
the use of myoblasts or satellite cells to treat DMD in the clinic has not been realized yet. …
the use of myoblasts or satellite cells to treat DMD in the clinic has not been realized yet. …
Concordant but varied phenotypes among Duchenne muscular dystrophy patient-specific myoblasts derived using a human iPSC-based model
IY Choi, HT Lim, K Estrellas, J Mula, TV Cohen… - Cell reports, 2016 - cell.com
… fully applied to the hiPSCs of muscular dystrophy, mainly due to the absence of a successful
strategy for isolating expandable functional myoblasts. Previous efforts to derive myogenic …
strategy for isolating expandable functional myoblasts. Previous efforts to derive myogenic …
Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy
JN Robinson-Hamm, CA Gersbach - Human genetics, 2016 - Springer
… Genome editing of the dystrophin gene has been successful in cultured myoblasts, induced
pluripotent stem cells, and fibroblasts with zinc finger nucleases, TALENs, meganucleases, …
pluripotent stem cells, and fibroblasts with zinc finger nucleases, TALENs, meganucleases, …
Restoring dystrophin expression in Duchenne muscular dystrophy: current status of therapeutic approaches
Y Shimizu-Motohashi, H Komaki, N Motohashi… - Journal of personalized …, 2019 - mdpi.com
… Duchenne muscular dystrophy (DMD) is a muscular disorder … reduction of the muscle
cytoskeletal protein dystrophin. The … into myoblasts upon injury to generate new muscle fibers [69]…
cytoskeletal protein dystrophin. The … into myoblasts upon injury to generate new muscle fibers [69]…
Placenta-derived mesenchymal stromal cells and their exosomes exert therapeutic effects in Duchenne muscular dystrophy
A Bier, P Berenstein, N Kronfeld, D Morgoulis, A Ziv-Av… - Biomaterials, 2018 - Elsevier
… human myoblasts from healthy controls, Duchenne patients and mdx mice. Treatment of
myoblasts … To demonstrate that the transferred miR-29c played a role in the increased myoblast …
myoblasts … To demonstrate that the transferred miR-29c played a role in the increased myoblast …