Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies

Wal, Erik van der; Herrero-Hernandez, Pablo; Wan, Raymond; Broeders, Mike; Groen, Stijn L.M. in ‘t; Gestel, Tom J.M. van; IJcken, Wilfred F. J. van; Cheung, Tom H.; Ploeg, Ans T. van der; Schaaf, Gerben; Pijnappel, W.W.M. Pim

doi:10.1016/j.stemcr.2018.04.002

Cited by 92 publications

(94 citation statements)

References 35 publications

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“…Given these findings we propose that the ESM model may represent a versatile tool to (1) further dissect satellite cell biology, (2) establish skeletal muscle disease models in vitro for drug development, and (3) provide potential therapeutic satellite cells for cellbased skeletal muscle repair. The availability of human pluripotent stem cells and methods for their directed myoblast differentiation 58,59 will facilitate the translation of the reported rat to a human model. In fact, alternative models of engineered human skeletal muscle using the forced expression of Pax7 60 or directed multi-lineage differentiation in induced pluripotent stem cells 61 have recently been reported.…”

Section: Discussionmentioning

confidence: 99%

Regeneration competent satellite cell niches in rat engineered skeletal muscle

et al. 2019

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Satellite cells reside in defined niches and are activated upon skeletal muscle injury to facilitate regeneration. Mechanistic studies of skeletal muscle regeneration are hampered by the inability to faithfully simulate satellite cell biology in vitro. We sought to overcome this limitation by developing tissue engineered skeletal muscle (ESM) with (1) satellite cell niches and (2) the capacity to regenerate after injury. ESMs contained quiescent Pax7‐positive satellite cells in morphologically defined niches. Satellite cells could be activated to repair (i) cardiotoxin and (ii) mechanical crush injuries. Activation of the Wnt‐pathway was essential for muscle regeneration. Finally, muscle progenitors from the engineered niche developed de novo ESM in vitro and regenerated skeletal muscle after cardiotoxin‐induced injury in vivo. We conclude that ESM with functional progenitor niches reminiscent of the in vivo satellite cell niches can be engineered in vitro. ESM may ultimately be exploited in disease modeling, drug screening, or muscle regeneration.

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Section: Discussionmentioning

confidence: 99%

Regeneration competent satellite cell niches in rat engineered skeletal muscle

et al. 2019

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“…hiPSCs can be generated from the patient's somatic cells, and transplantation of hiPSC-derived cells do not induce immune rejection in the patient as in heterologous transplantation [155]. Furthermore, gene mutation can be corrected by genome editing technology in patient-derived iPSCs [159], and gene-corrected iPSC-derived cells may be ideal cell sources for regenerative therapy. In DMD, innovation is still necessary to develop the method to deliver the cells to whole body.…”

Section: Regenerative Strategymentioning

confidence: 99%

Stem Cell Aging in Skeletal Muscle Regeneration and Disease

Yamakawa

Kusumoto

Hashimoto

et al. 2020

IJMS

100

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Skeletal muscle comprises 30–40% of the weight of a healthy human body and is required for voluntary movements in humans. Mature skeletal muscle is formed by multinuclear cells, which are called myofibers. Formation of myofibers depends on the proliferation, differentiation, and fusion of muscle progenitor cells during development and after injury. Muscle progenitor cells are derived from muscle satellite (stem) cells (MuSCs), which reside on the surface of the myofiber but beneath the basement membrane. MuSCs play a central role in postnatal maintenance, growth, repair, and regeneration of skeletal muscle. In sedentary adult muscle, MuSCs are mitotically quiescent, but are promptly activated in response to muscle injury. Physiological and chronological aging induces MuSC aging, leading to an impaired regenerative capability. Importantly, in pathological situations, repetitive muscle injury induces early impairment of MuSCs due to stem cell aging and leads to early impairment of regeneration ability. In this review, we discuss (1) the role of MuSCs in muscle regeneration, (2) stem cell aging under physiological and pathological conditions, and (3) prospects related to clinical applications of controlling MuSCs.

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“…34,35 Correction of mutations in DMD cells could be achieved by CT followed by differentiation to muscle cells and their injection in the muscles, an option that has been used by several researchers in muscle pathology. 27,33,[36][37][38] The only needed additional step to the protocol used herein is the inactivation, before retro-MMCT fusion, of the HPRT gene on the endogenous X chromosome by CRISPR technology, allowing the use of HAT selection after retro-MMCT. We have recently shown the feasibility of this step by correcting the genetic defect in iPSCs from a mouse model of the X-linked CGD.…”

Section: Discussionmentioning

confidence: 99%

Chromosome Transplantation: A Possible Approach to Treat Human X-linked Disorders

Paulis

Susani

Castelli

et al. 2020

Molecular Therapy - Methods & Clinical Development

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Many human genetic diseases are associated with gross mutations such as aneuploidies, deletions, duplications, or inversions. For these "structural" disorders, conventional gene therapy, based on viral vectors and/or on programmable nuclease-mediated homologous recombination, is still unsatisfactory. To correct such disorders, chromosome transplantation (CT), defined as the perfect substitution of an endogenous defective chromosome with an exogenous normal one, could be applied. CT re-establishes a normal diploid cell, leaving no marker of the procedure, as we have recently shown in mouse pluripotent stem cells. To prove the feasibility of the CT approach in human cells, we used human induced pluripotent stem cells (hiPSCs) reprogrammed from Lesch-Nyhan (LN) disease patients, taking advantage of their mutation in the X-linked HPRT gene, making the LN cells selectable and distinguishable from the resistant corrected normal cells. In this study, we demonstrate, for the first time, that CT is feasible in hiPSCs: the normal exogenous X chromosome was first transferred using an improved chromosome transfer system, and the extra sex chromosome was spontaneously lost. These CT cells were functionally corrected and maintained their pluripotency and differentiation capability. By inactivation of the autologous HPRT gene, CT paves the way to the correction of hiPSCs from several X-linked disorders.

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Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies

Cited by 92 publications

References 35 publications

Regeneration competent satellite cell niches in rat engineered skeletal muscle

Regeneration competent satellite cell niches in rat engineered skeletal muscle

Stem Cell Aging in Skeletal Muscle Regeneration and Disease

Chromosome Transplantation: A Possible Approach to Treat Human X-linked Disorders

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