Generation of allogeneic and xenogeneic functional muscle stem cells for intramuscular transplantation
Satellite cells, the stem cells of skeletal muscle tissue, hold a remarkable regeneration capacity and therapeutic potential in regenerative medicine. However, low satellite cell yield from autologous or donor-derived muscles hinders the adoption of satellite cell transplantation for the treatment o...
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| Veröffentlicht in: | The Journal of clinical investigation 2024-06, Vol.134 (12) |
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| creator | Lenardic, Ajda Domenig, Seraina A Zvick, Joel Bundschuh, Nicola Tarnowska- Sengul, Monika Furrer, Regula Noe, Falko Trautmann, Christine L Ghosh, Adhideb Bacchin, Giada Gjonlleshaj, Pjeter Qabrati, Xhem Masschelein, Evi De Bock, Katrien Handschin, Christoph Bar-Nur, Ori |
| description | Satellite cells, the stem cells of skeletal muscle tissue, hold a remarkable regeneration capacity and therapeutic potential in regenerative medicine. However, low satellite cell yield from autologous or donor-derived muscles hinders the adoption of satellite cell transplantation for the treatment of muscle diseases, including Duchenne muscular dystrophy (DMD). To address this limitation, here we investigated whether satellite cells can be derived in allogeneic or xenogeneic animal hosts. First, injection of CRISPR/Cas9-corrected [Dmd.sup.mdx] mouse induced pluripotent stem cells (iPSCs) into mouse blastocysts carrying an ablation system of host satellite cells gave rise to intraspecies chimeras exclusively carrying iPSC-derived satellite cells. Furthermore, injection of genetically corrected DMD iPSCs into rat blastocysts resulted in the formation of interspecies rat-mouse chimeras harboring mouse satellite cells. Notably, iPSC-derived satellite cells or derivative myoblasts produced in intraspecies or interspecies chimeras restored dystrophin expression in DMD mice following intramuscular transplantation and contributed to the satellite cell pool. Collectively, this study demonstrates the feasibility of producing therapeutically competent stem cells across divergent animal species, raising the possibility of generating human muscle stem cells in large animals for regenerative medicine purposes. |
| doi_str_mv | 10.1172/JCI166998 |
| format | Article |
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However, low satellite cell yield from autologous or donor-derived muscles hinders the adoption of satellite cell transplantation for the treatment of muscle diseases, including Duchenne muscular dystrophy (DMD). To address this limitation, here we investigated whether satellite cells can be derived in allogeneic or xenogeneic animal hosts. First, injection of CRISPR/Cas9-corrected [Dmd.sup.mdx] mouse induced pluripotent stem cells (iPSCs) into mouse blastocysts carrying an ablation system of host satellite cells gave rise to intraspecies chimeras exclusively carrying iPSC-derived satellite cells. Furthermore, injection of genetically corrected DMD iPSCs into rat blastocysts resulted in the formation of interspecies rat-mouse chimeras harboring mouse satellite cells. Notably, iPSC-derived satellite cells or derivative myoblasts produced in intraspecies or interspecies chimeras restored dystrophin expression in DMD mice following intramuscular transplantation and contributed to the satellite cell pool. Collectively, this study demonstrates the feasibility of producing therapeutically competent stem cells across divergent animal species, raising the possibility of generating human muscle stem cells in large animals for regenerative medicine purposes.</description><identifier>ISSN: 1558-8238</identifier><identifier>ISSN: 0021-9738</identifier><identifier>EISSN: 1558-8238</identifier><identifier>DOI: 10.1172/JCI166998</identifier><identifier>PMID: 38713532</identifier><language>eng</language><publisher>American Society for Clinical Investigation</publisher><subject>Care and treatment ; Degeneration (Pathology) ; Duchenne muscular dystrophy ; Dystrophin ; Genetic aspects ; Health aspects ; Muscles ; Physiological aspects ; Regenerative medicine ; Stem cell research ; Stem cells ; Transplantation ; Utrophin</subject><ispartof>The Journal of clinical investigation, 2024-06, Vol.134 (12)</ispartof><rights>COPYRIGHT 2024 American Society for Clinical Investigation</rights><rights>2024 Lenardič et al. 2024 Lenardič et al.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><cites>FETCH-LOGICAL-c447t-d62fe3ef83c0ab338f7276960a5c6436f9c737bb96f2d1739440f07a82b9a3483</cites><orcidid>0000-0001-8232-4663 ; 0000-0002-5160-4571</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC11178549/pdf/$$EPDF$$P50$$Gpubmedcentral$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC11178549/$$EHTML$$P50$$Gpubmedcentral$$Hfree_for_read</linktohtml><link.rule.ids>230,314,723,776,780,881,27903,27904,53770,53772</link.rule.ids></links><search><creatorcontrib>Lenardic, Ajda</creatorcontrib><creatorcontrib>Domenig, Seraina A</creatorcontrib><creatorcontrib>Zvick, Joel</creatorcontrib><creatorcontrib>Bundschuh, Nicola</creatorcontrib><creatorcontrib>Tarnowska- Sengul, Monika</creatorcontrib><creatorcontrib>Furrer, Regula</creatorcontrib><creatorcontrib>Noe, Falko</creatorcontrib><creatorcontrib>Trautmann, Christine L</creatorcontrib><creatorcontrib>Ghosh, Adhideb</creatorcontrib><creatorcontrib>Bacchin, Giada</creatorcontrib><creatorcontrib>Gjonlleshaj, Pjeter</creatorcontrib><creatorcontrib>Qabrati, Xhem</creatorcontrib><creatorcontrib>Masschelein, Evi</creatorcontrib><creatorcontrib>De Bock, Katrien</creatorcontrib><creatorcontrib>Handschin, Christoph</creatorcontrib><creatorcontrib>Bar-Nur, Ori</creatorcontrib><title>Generation of allogeneic and xenogeneic functional muscle stem cells for intramuscular transplantation</title><title>The Journal of clinical investigation</title><description>Satellite cells, the stem cells of skeletal muscle tissue, hold a remarkable regeneration capacity and therapeutic potential in regenerative medicine. However, low satellite cell yield from autologous or donor-derived muscles hinders the adoption of satellite cell transplantation for the treatment of muscle diseases, including Duchenne muscular dystrophy (DMD). To address this limitation, here we investigated whether satellite cells can be derived in allogeneic or xenogeneic animal hosts. First, injection of CRISPR/Cas9-corrected [Dmd.sup.mdx] mouse induced pluripotent stem cells (iPSCs) into mouse blastocysts carrying an ablation system of host satellite cells gave rise to intraspecies chimeras exclusively carrying iPSC-derived satellite cells. Furthermore, injection of genetically corrected DMD iPSCs into rat blastocysts resulted in the formation of interspecies rat-mouse chimeras harboring mouse satellite cells. Notably, iPSC-derived satellite cells or derivative myoblasts produced in intraspecies or interspecies chimeras restored dystrophin expression in DMD mice following intramuscular transplantation and contributed to the satellite cell pool. Collectively, this study demonstrates the feasibility of producing therapeutically competent stem cells across divergent animal species, raising the possibility of generating human muscle stem cells in large animals for regenerative medicine purposes.</description><subject>Care and treatment</subject><subject>Degeneration (Pathology)</subject><subject>Duchenne muscular dystrophy</subject><subject>Dystrophin</subject><subject>Genetic aspects</subject><subject>Health aspects</subject><subject>Muscles</subject><subject>Physiological aspects</subject><subject>Regenerative medicine</subject><subject>Stem cell research</subject><subject>Stem cells</subject><subject>Transplantation</subject><subject>Utrophin</subject><issn>1558-8238</issn><issn>0021-9738</issn><issn>1558-8238</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2024</creationdate><recordtype>article</recordtype><recordid>eNqNkk1v3CAQhq2qVZOmPfQfIFWq2oMTMGDDqYpWSbpVpEj9uiKMBy8Vhq2xq-TfBydplJX2EHFgmHnmFbxMUbwn-JiQpjr5tlqTupZSvCgOCeeiFBUVL5_EB8WblP5gTBjj7HVxQEVDKKfVYWEvIMCoJxcDihZp72OfM84gHTp0DeH_0c7BLJT2aJiT8YDSBAMy4H1CNo7IhWnUS2n2ekQ5DmnrdZjutN8Wr6z2Cd497EfFr_Ozn6uv5eXVxXp1elkaxpqp7OrKAgUrqMG6pVTYpmpqWWPNTc1obaVpaNO2srZVRxoqGcMWN1pUrdSUCXpUfLnX3c7tAJ2B5VJebUc36PFGRe3UbiW4jerjP0WykYIzmRU-PSiM8e8MaVKDS8srdYA4J0Uxr7hktWAZ_XCP9tqDcsHGLGkWXJ0KTCQnmPFMlXuo_s52HwNYl9M7_PEePq8OBmf2NnzeacjMBNdTr-eU1PrH9-ezV7932Y9P2A1oP21S9PPyn2mvqBljSiPYR78JVsuAqscBpbc3kNRQ</recordid><startdate>20240615</startdate><enddate>20240615</enddate><creator>Lenardic, Ajda</creator><creator>Domenig, Seraina A</creator><creator>Zvick, Joel</creator><creator>Bundschuh, Nicola</creator><creator>Tarnowska- Sengul, Monika</creator><creator>Furrer, Regula</creator><creator>Noe, Falko</creator><creator>Trautmann, Christine L</creator><creator>Ghosh, Adhideb</creator><creator>Bacchin, Giada</creator><creator>Gjonlleshaj, Pjeter</creator><creator>Qabrati, Xhem</creator><creator>Masschelein, Evi</creator><creator>De Bock, Katrien</creator><creator>Handschin, Christoph</creator><creator>Bar-Nur, Ori</creator><general>American Society for Clinical Investigation</general><scope>AAYXX</scope><scope>CITATION</scope><scope>IOV</scope><scope>ISR</scope><scope>7X8</scope><scope>5PM</scope><orcidid>https://orcid.org/0000-0001-8232-4663</orcidid><orcidid>https://orcid.org/0000-0002-5160-4571</orcidid></search><sort><creationdate>20240615</creationdate><title>Generation of allogeneic and xenogeneic functional muscle stem cells for intramuscular transplantation</title><author>Lenardic, Ajda ; Domenig, Seraina A ; Zvick, Joel ; Bundschuh, Nicola ; Tarnowska- Sengul, Monika ; Furrer, Regula ; Noe, Falko ; Trautmann, Christine L ; Ghosh, Adhideb ; Bacchin, Giada ; Gjonlleshaj, Pjeter ; Qabrati, Xhem ; Masschelein, Evi ; De Bock, Katrien ; Handschin, Christoph ; Bar-Nur, Ori</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c447t-d62fe3ef83c0ab338f7276960a5c6436f9c737bb96f2d1739440f07a82b9a3483</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2024</creationdate><topic>Care and treatment</topic><topic>Degeneration (Pathology)</topic><topic>Duchenne muscular dystrophy</topic><topic>Dystrophin</topic><topic>Genetic aspects</topic><topic>Health aspects</topic><topic>Muscles</topic><topic>Physiological aspects</topic><topic>Regenerative medicine</topic><topic>Stem cell research</topic><topic>Stem cells</topic><topic>Transplantation</topic><topic>Utrophin</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Lenardic, Ajda</creatorcontrib><creatorcontrib>Domenig, Seraina A</creatorcontrib><creatorcontrib>Zvick, Joel</creatorcontrib><creatorcontrib>Bundschuh, Nicola</creatorcontrib><creatorcontrib>Tarnowska- Sengul, Monika</creatorcontrib><creatorcontrib>Furrer, Regula</creatorcontrib><creatorcontrib>Noe, Falko</creatorcontrib><creatorcontrib>Trautmann, Christine L</creatorcontrib><creatorcontrib>Ghosh, Adhideb</creatorcontrib><creatorcontrib>Bacchin, Giada</creatorcontrib><creatorcontrib>Gjonlleshaj, Pjeter</creatorcontrib><creatorcontrib>Qabrati, Xhem</creatorcontrib><creatorcontrib>Masschelein, Evi</creatorcontrib><creatorcontrib>De Bock, Katrien</creatorcontrib><creatorcontrib>Handschin, Christoph</creatorcontrib><creatorcontrib>Bar-Nur, Ori</creatorcontrib><collection>CrossRef</collection><collection>Gale In Context: Opposing Viewpoints</collection><collection>Gale In Context: Science</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>The Journal of clinical investigation</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Lenardic, Ajda</au><au>Domenig, Seraina A</au><au>Zvick, Joel</au><au>Bundschuh, Nicola</au><au>Tarnowska- Sengul, Monika</au><au>Furrer, Regula</au><au>Noe, Falko</au><au>Trautmann, Christine L</au><au>Ghosh, Adhideb</au><au>Bacchin, Giada</au><au>Gjonlleshaj, Pjeter</au><au>Qabrati, Xhem</au><au>Masschelein, Evi</au><au>De Bock, Katrien</au><au>Handschin, Christoph</au><au>Bar-Nur, Ori</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Generation of allogeneic and xenogeneic functional muscle stem cells for intramuscular transplantation</atitle><jtitle>The Journal of clinical investigation</jtitle><date>2024-06-15</date><risdate>2024</risdate><volume>134</volume><issue>12</issue><issn>1558-8238</issn><issn>0021-9738</issn><eissn>1558-8238</eissn><abstract>Satellite cells, the stem cells of skeletal muscle tissue, hold a remarkable regeneration capacity and therapeutic potential in regenerative medicine. However, low satellite cell yield from autologous or donor-derived muscles hinders the adoption of satellite cell transplantation for the treatment of muscle diseases, including Duchenne muscular dystrophy (DMD). To address this limitation, here we investigated whether satellite cells can be derived in allogeneic or xenogeneic animal hosts. First, injection of CRISPR/Cas9-corrected [Dmd.sup.mdx] mouse induced pluripotent stem cells (iPSCs) into mouse blastocysts carrying an ablation system of host satellite cells gave rise to intraspecies chimeras exclusively carrying iPSC-derived satellite cells. Furthermore, injection of genetically corrected DMD iPSCs into rat blastocysts resulted in the formation of interspecies rat-mouse chimeras harboring mouse satellite cells. Notably, iPSC-derived satellite cells or derivative myoblasts produced in intraspecies or interspecies chimeras restored dystrophin expression in DMD mice following intramuscular transplantation and contributed to the satellite cell pool. Collectively, this study demonstrates the feasibility of producing therapeutically competent stem cells across divergent animal species, raising the possibility of generating human muscle stem cells in large animals for regenerative medicine purposes.</abstract><pub>American Society for Clinical Investigation</pub><pmid>38713532</pmid><doi>10.1172/JCI166998</doi><orcidid>https://orcid.org/0000-0001-8232-4663</orcidid><orcidid>https://orcid.org/0000-0002-5160-4571</orcidid><oa>free_for_read</oa></addata></record> |
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| subjects | Care and treatment Degeneration (Pathology) Duchenne muscular dystrophy Dystrophin Genetic aspects Health aspects Muscles Physiological aspects Regenerative medicine Stem cell research Stem cells Transplantation Utrophin |
| title | Generation of allogeneic and xenogeneic functional muscle stem cells for intramuscular transplantation |
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