Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)
Mucopolysaccharidosis type I (MPS IH; Hurler syndrome) is a congenital deficiency of α-L-iduronidase, leading to lysosomal storage of glycosaminoglycans that is ultimately fatal following an insidious onset after birth. Hematopoietic cell transplantation (HCT) is a life-saving measure in MPS IH. How...
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| Veröffentlicht in: | Blood 2011-01, Vol.117 (3), p.839-847 |
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| creator | Tolar, Jakub Park, In-Hyun Xia, Lily Lees, Chris J. Peacock, Brandon Webber, Beau McElmurry, Ron T. Eide, Cindy R. Orchard, Paul J. Kyba, Michael Osborn, Mark J. Lund, Troy C. Wagner, John E. Daley, George Q. Blazar, Bruce R. |
| description | Mucopolysaccharidosis type I (MPS IH; Hurler syndrome) is a congenital deficiency of α-L-iduronidase, leading to lysosomal storage of glycosaminoglycans that is ultimately fatal following an insidious onset after birth. Hematopoietic cell transplantation (HCT) is a life-saving measure in MPS IH. However, because a suitable hematopoietic donor is not found for everyone, because HCT is associated with significant morbidity and mortality, and because there is no known benefit of immune reaction between the host and the donor cells in MPS IH, gene-corrected autologous stem cells may be the ideal graft for HCT. Thus, we generated induced pluripotent stem cells from 2 patients with MPS IH (MPS-iPS cells). We found that α-L-iduronidase was not required for stem cell renewal, and that MPS-iPS cells showed lysosomal storage characteristic of MPS IH and could be differentiated to both hematopoietic and nonhematopoietic cells. The specific epigenetic profile associated with de-differentiation of MPS IH fibroblasts into MPS-iPS cells was maintained when MPS-iPS cells are gene-corrected with virally delivered α-L-iduronidase. These data underscore the potential of MPS-iPS cells to generate autologous hematopoietic grafts devoid of immunologic complications of allogeneic transplantation, as well as generating nonhematopoietic cells with the potential to treat anatomical sites not fully corrected with HCT. |
| doi_str_mv | 10.1182/blood-2010-05-287607 |
| format | Article |
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Hematopoietic cell transplantation (HCT) is a life-saving measure in MPS IH. However, because a suitable hematopoietic donor is not found for everyone, because HCT is associated with significant morbidity and mortality, and because there is no known benefit of immune reaction between the host and the donor cells in MPS IH, gene-corrected autologous stem cells may be the ideal graft for HCT. Thus, we generated induced pluripotent stem cells from 2 patients with MPS IH (MPS-iPS cells). We found that α-L-iduronidase was not required for stem cell renewal, and that MPS-iPS cells showed lysosomal storage characteristic of MPS IH and could be differentiated to both hematopoietic and nonhematopoietic cells. The specific epigenetic profile associated with de-differentiation of MPS IH fibroblasts into MPS-iPS cells was maintained when MPS-iPS cells are gene-corrected with virally delivered α-L-iduronidase. These data underscore the potential of MPS-iPS cells to generate autologous hematopoietic grafts devoid of immunologic complications of allogeneic transplantation, as well as generating nonhematopoietic cells with the potential to treat anatomical sites not fully corrected with HCT.</description><identifier>ISSN: 0006-4971</identifier><identifier>EISSN: 1528-0020</identifier><identifier>DOI: 10.1182/blood-2010-05-287607</identifier><identifier>PMID: 21037085</identifier><language>eng</language><publisher>Washington, DC: Elsevier Inc</publisher><subject>Animals ; Biological and medical sciences ; Bone Marrow Cells - cytology ; Bone Marrow Cells - metabolism ; Carbohydrates (enzymatic deficiencies). Glycogenosis ; Cell Differentiation ; Cells, Cultured ; Child, Preschool ; DNA Methylation ; Errors of metabolism ; HEK293 Cells ; Hematologic and hematopoietic diseases ; Hematopoiesis and Stem Cells ; Hematopoietic System - cytology ; Hematopoietic System - metabolism ; Homeodomain Proteins - genetics ; Homeodomain Proteins - metabolism ; Humans ; Iduronidase - genetics ; Iduronidase - metabolism ; Induced Pluripotent Stem Cells - cytology ; Induced Pluripotent Stem Cells - metabolism ; Infant ; Keratinocytes - cytology ; Keratinocytes - metabolism ; Kruppel-Like Transcription Factors - genetics ; Kruppel-Like Transcription Factors - metabolism ; Male ; Medical sciences ; Mesoderm - cytology ; Mesoderm - metabolism ; Metabolic diseases ; Mice ; Mucopolysaccharidosis I - genetics ; Mucopolysaccharidosis I - metabolism ; Mucopolysaccharidosis I - pathology ; Nanog Homeobox Protein ; Octamer Transcription Factor-3 - genetics ; Octamer Transcription Factor-3 - metabolism ; Promoter Regions, Genetic - genetics ; Proto-Oncogene Proteins c-myc - genetics ; Proto-Oncogene Proteins c-myc - metabolism ; SOXB1 Transcription Factors - genetics ; SOXB1 Transcription Factors - metabolism ; Stromal Cells - cytology ; Stromal Cells - metabolism ; Transfection</subject><ispartof>Blood, 2011-01, Vol.117 (3), p.839-847</ispartof><rights>2011 American Society of Hematology</rights><rights>2015 INIST-CNRS</rights><rights>2011 by The American Society of Hematology</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c492t-9b2f877cb95fb2bb4778180d13a721b806a1025ef6f1247677e8ab77c5e3b20d3</citedby><cites>FETCH-LOGICAL-c492t-9b2f877cb95fb2bb4778180d13a721b806a1025ef6f1247677e8ab77c5e3b20d3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>230,314,780,784,885,27924,27925</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=23791208$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/21037085$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Tolar, Jakub</creatorcontrib><creatorcontrib>Park, In-Hyun</creatorcontrib><creatorcontrib>Xia, Lily</creatorcontrib><creatorcontrib>Lees, Chris J.</creatorcontrib><creatorcontrib>Peacock, Brandon</creatorcontrib><creatorcontrib>Webber, Beau</creatorcontrib><creatorcontrib>McElmurry, Ron T.</creatorcontrib><creatorcontrib>Eide, Cindy R.</creatorcontrib><creatorcontrib>Orchard, Paul J.</creatorcontrib><creatorcontrib>Kyba, Michael</creatorcontrib><creatorcontrib>Osborn, Mark J.</creatorcontrib><creatorcontrib>Lund, Troy C.</creatorcontrib><creatorcontrib>Wagner, John E.</creatorcontrib><creatorcontrib>Daley, George Q.</creatorcontrib><creatorcontrib>Blazar, Bruce R.</creatorcontrib><title>Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)</title><title>Blood</title><addtitle>Blood</addtitle><description>Mucopolysaccharidosis type I (MPS IH; Hurler syndrome) is a congenital deficiency of α-L-iduronidase, leading to lysosomal storage of glycosaminoglycans that is ultimately fatal following an insidious onset after birth. Hematopoietic cell transplantation (HCT) is a life-saving measure in MPS IH. However, because a suitable hematopoietic donor is not found for everyone, because HCT is associated with significant morbidity and mortality, and because there is no known benefit of immune reaction between the host and the donor cells in MPS IH, gene-corrected autologous stem cells may be the ideal graft for HCT. Thus, we generated induced pluripotent stem cells from 2 patients with MPS IH (MPS-iPS cells). We found that α-L-iduronidase was not required for stem cell renewal, and that MPS-iPS cells showed lysosomal storage characteristic of MPS IH and could be differentiated to both hematopoietic and nonhematopoietic cells. The specific epigenetic profile associated with de-differentiation of MPS IH fibroblasts into MPS-iPS cells was maintained when MPS-iPS cells are gene-corrected with virally delivered α-L-iduronidase. These data underscore the potential of MPS-iPS cells to generate autologous hematopoietic grafts devoid of immunologic complications of allogeneic transplantation, as well as generating nonhematopoietic cells with the potential to treat anatomical sites not fully corrected with HCT.</description><subject>Animals</subject><subject>Biological and medical sciences</subject><subject>Bone Marrow Cells - cytology</subject><subject>Bone Marrow Cells - metabolism</subject><subject>Carbohydrates (enzymatic deficiencies). Glycogenosis</subject><subject>Cell Differentiation</subject><subject>Cells, Cultured</subject><subject>Child, Preschool</subject><subject>DNA Methylation</subject><subject>Errors of metabolism</subject><subject>HEK293 Cells</subject><subject>Hematologic and hematopoietic diseases</subject><subject>Hematopoiesis and Stem Cells</subject><subject>Hematopoietic System - cytology</subject><subject>Hematopoietic System - metabolism</subject><subject>Homeodomain Proteins - genetics</subject><subject>Homeodomain Proteins - metabolism</subject><subject>Humans</subject><subject>Iduronidase - genetics</subject><subject>Iduronidase - metabolism</subject><subject>Induced Pluripotent Stem Cells - cytology</subject><subject>Induced Pluripotent Stem Cells - metabolism</subject><subject>Infant</subject><subject>Keratinocytes - cytology</subject><subject>Keratinocytes - metabolism</subject><subject>Kruppel-Like Transcription Factors - genetics</subject><subject>Kruppel-Like Transcription Factors - metabolism</subject><subject>Male</subject><subject>Medical sciences</subject><subject>Mesoderm - cytology</subject><subject>Mesoderm - metabolism</subject><subject>Metabolic diseases</subject><subject>Mice</subject><subject>Mucopolysaccharidosis I - genetics</subject><subject>Mucopolysaccharidosis I - metabolism</subject><subject>Mucopolysaccharidosis I - pathology</subject><subject>Nanog Homeobox Protein</subject><subject>Octamer Transcription Factor-3 - genetics</subject><subject>Octamer Transcription Factor-3 - metabolism</subject><subject>Promoter Regions, Genetic - genetics</subject><subject>Proto-Oncogene Proteins c-myc - genetics</subject><subject>Proto-Oncogene Proteins c-myc - metabolism</subject><subject>SOXB1 Transcription Factors - genetics</subject><subject>SOXB1 Transcription Factors - metabolism</subject><subject>Stromal Cells - cytology</subject><subject>Stromal Cells - metabolism</subject><subject>Transfection</subject><issn>0006-4971</issn><issn>1528-0020</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2011</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp9kUtv1DAUhSMEokPhHyDkDQIWgWvnYWeDhCpgKlViA2vLj2vGKImD7RTNsv-8HmZoYcPKku85x8f3q6rnFN5SKtg7PYZgawYUauhqJngP_EG1oR0TNQCDh9UGAPq6HTg9q56k9AOAtg3rHldnjELDQXSb6maLk8phCR6zN8R65zDinL3KPswkOOJnuxq0ZBnX6JeQy5CkjBMxOI6JuBgmshR1uU_kl887Mq2mBI77pIzZqehtSD6RvF-QXJLX2zWOGEnaz7ZY8c3T6pFTY8Jnp_O8-vbp49eLbX315fPlxYer2rQDy_WgmROcGz10TjOtW84FFWBpozijWkCvKLAOXe8oa3nPOQqli6HDRjOwzXn1_pi7rHpCa0rfqEa5RD-puJdBefnvZPY7-T1cywaaDjgvAa9OATH8XDFlOfl0WIKaMaxJirYXwzD0oijbo9LEkFJEd_cKBXmAJ3_Dkwd4Ejp5hFdsL_5ueGf6Q6sIXp4EKhk1uqhm49O9ruEDZSDuv4pln9ceo0ym8CkUfUSTpQ3-_01uAe-avMM</recordid><startdate>20110120</startdate><enddate>20110120</enddate><creator>Tolar, Jakub</creator><creator>Park, In-Hyun</creator><creator>Xia, Lily</creator><creator>Lees, Chris J.</creator><creator>Peacock, Brandon</creator><creator>Webber, Beau</creator><creator>McElmurry, Ron T.</creator><creator>Eide, Cindy R.</creator><creator>Orchard, Paul J.</creator><creator>Kyba, Michael</creator><creator>Osborn, Mark J.</creator><creator>Lund, Troy C.</creator><creator>Wagner, John E.</creator><creator>Daley, George Q.</creator><creator>Blazar, Bruce R.</creator><general>Elsevier Inc</general><general>Americain Society of Hematology</general><general>American Society of Hematology</general><scope>6I.</scope><scope>AAFTH</scope><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>5PM</scope></search><sort><creationdate>20110120</creationdate><title>Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)</title><author>Tolar, Jakub ; Park, In-Hyun ; Xia, Lily ; Lees, Chris J. ; Peacock, Brandon ; Webber, Beau ; McElmurry, Ron T. ; Eide, Cindy R. ; Orchard, Paul J. ; Kyba, Michael ; Osborn, Mark J. ; Lund, Troy C. ; Wagner, John E. ; Daley, George Q. ; Blazar, Bruce R.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c492t-9b2f877cb95fb2bb4778180d13a721b806a1025ef6f1247677e8ab77c5e3b20d3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2011</creationdate><topic>Animals</topic><topic>Biological and medical sciences</topic><topic>Bone Marrow Cells - cytology</topic><topic>Bone Marrow Cells - metabolism</topic><topic>Carbohydrates (enzymatic deficiencies). Glycogenosis</topic><topic>Cell Differentiation</topic><topic>Cells, Cultured</topic><topic>Child, Preschool</topic><topic>DNA Methylation</topic><topic>Errors of metabolism</topic><topic>HEK293 Cells</topic><topic>Hematologic and hematopoietic diseases</topic><topic>Hematopoiesis and Stem Cells</topic><topic>Hematopoietic System - cytology</topic><topic>Hematopoietic System - metabolism</topic><topic>Homeodomain Proteins - genetics</topic><topic>Homeodomain Proteins - metabolism</topic><topic>Humans</topic><topic>Iduronidase - genetics</topic><topic>Iduronidase - metabolism</topic><topic>Induced Pluripotent Stem Cells - cytology</topic><topic>Induced Pluripotent Stem Cells - metabolism</topic><topic>Infant</topic><topic>Keratinocytes - cytology</topic><topic>Keratinocytes - metabolism</topic><topic>Kruppel-Like Transcription Factors - genetics</topic><topic>Kruppel-Like Transcription Factors - metabolism</topic><topic>Male</topic><topic>Medical sciences</topic><topic>Mesoderm - cytology</topic><topic>Mesoderm - metabolism</topic><topic>Metabolic diseases</topic><topic>Mice</topic><topic>Mucopolysaccharidosis I - genetics</topic><topic>Mucopolysaccharidosis I - metabolism</topic><topic>Mucopolysaccharidosis I - pathology</topic><topic>Nanog Homeobox Protein</topic><topic>Octamer Transcription Factor-3 - genetics</topic><topic>Octamer Transcription Factor-3 - metabolism</topic><topic>Promoter Regions, Genetic - genetics</topic><topic>Proto-Oncogene Proteins c-myc - genetics</topic><topic>Proto-Oncogene Proteins c-myc - metabolism</topic><topic>SOXB1 Transcription Factors - genetics</topic><topic>SOXB1 Transcription Factors - metabolism</topic><topic>Stromal Cells - cytology</topic><topic>Stromal Cells - metabolism</topic><topic>Transfection</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Tolar, Jakub</creatorcontrib><creatorcontrib>Park, In-Hyun</creatorcontrib><creatorcontrib>Xia, Lily</creatorcontrib><creatorcontrib>Lees, Chris J.</creatorcontrib><creatorcontrib>Peacock, Brandon</creatorcontrib><creatorcontrib>Webber, Beau</creatorcontrib><creatorcontrib>McElmurry, Ron T.</creatorcontrib><creatorcontrib>Eide, Cindy R.</creatorcontrib><creatorcontrib>Orchard, Paul J.</creatorcontrib><creatorcontrib>Kyba, Michael</creatorcontrib><creatorcontrib>Osborn, Mark J.</creatorcontrib><creatorcontrib>Lund, Troy C.</creatorcontrib><creatorcontrib>Wagner, John E.</creatorcontrib><creatorcontrib>Daley, George Q.</creatorcontrib><creatorcontrib>Blazar, Bruce R.</creatorcontrib><collection>ScienceDirect Open Access Titles</collection><collection>Elsevier:ScienceDirect:Open Access</collection><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Blood</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Tolar, Jakub</au><au>Park, In-Hyun</au><au>Xia, Lily</au><au>Lees, Chris J.</au><au>Peacock, Brandon</au><au>Webber, Beau</au><au>McElmurry, Ron T.</au><au>Eide, Cindy R.</au><au>Orchard, Paul J.</au><au>Kyba, Michael</au><au>Osborn, Mark J.</au><au>Lund, Troy C.</au><au>Wagner, John E.</au><au>Daley, George Q.</au><au>Blazar, Bruce R.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)</atitle><jtitle>Blood</jtitle><addtitle>Blood</addtitle><date>2011-01-20</date><risdate>2011</risdate><volume>117</volume><issue>3</issue><spage>839</spage><epage>847</epage><pages>839-847</pages><issn>0006-4971</issn><eissn>1528-0020</eissn><abstract>Mucopolysaccharidosis type I (MPS IH; Hurler syndrome) is a congenital deficiency of α-L-iduronidase, leading to lysosomal storage of glycosaminoglycans that is ultimately fatal following an insidious onset after birth. Hematopoietic cell transplantation (HCT) is a life-saving measure in MPS IH. However, because a suitable hematopoietic donor is not found for everyone, because HCT is associated with significant morbidity and mortality, and because there is no known benefit of immune reaction between the host and the donor cells in MPS IH, gene-corrected autologous stem cells may be the ideal graft for HCT. Thus, we generated induced pluripotent stem cells from 2 patients with MPS IH (MPS-iPS cells). We found that α-L-iduronidase was not required for stem cell renewal, and that MPS-iPS cells showed lysosomal storage characteristic of MPS IH and could be differentiated to both hematopoietic and nonhematopoietic cells. The specific epigenetic profile associated with de-differentiation of MPS IH fibroblasts into MPS-iPS cells was maintained when MPS-iPS cells are gene-corrected with virally delivered α-L-iduronidase. These data underscore the potential of MPS-iPS cells to generate autologous hematopoietic grafts devoid of immunologic complications of allogeneic transplantation, as well as generating nonhematopoietic cells with the potential to treat anatomical sites not fully corrected with HCT.</abstract><cop>Washington, DC</cop><pub>Elsevier Inc</pub><pmid>21037085</pmid><doi>10.1182/blood-2010-05-287607</doi><tpages>9</tpages><oa>free_for_read</oa></addata></record> |
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| subjects | Animals Biological and medical sciences Bone Marrow Cells - cytology Bone Marrow Cells - metabolism Carbohydrates (enzymatic deficiencies). Glycogenosis Cell Differentiation Cells, Cultured Child, Preschool DNA Methylation Errors of metabolism HEK293 Cells Hematologic and hematopoietic diseases Hematopoiesis and Stem Cells Hematopoietic System - cytology Hematopoietic System - metabolism Homeodomain Proteins - genetics Homeodomain Proteins - metabolism Humans Iduronidase - genetics Iduronidase - metabolism Induced Pluripotent Stem Cells - cytology Induced Pluripotent Stem Cells - metabolism Infant Keratinocytes - cytology Keratinocytes - metabolism Kruppel-Like Transcription Factors - genetics Kruppel-Like Transcription Factors - metabolism Male Medical sciences Mesoderm - cytology Mesoderm - metabolism Metabolic diseases Mice Mucopolysaccharidosis I - genetics Mucopolysaccharidosis I - metabolism Mucopolysaccharidosis I - pathology Nanog Homeobox Protein Octamer Transcription Factor-3 - genetics Octamer Transcription Factor-3 - metabolism Promoter Regions, Genetic - genetics Proto-Oncogene Proteins c-myc - genetics Proto-Oncogene Proteins c-myc - metabolism SOXB1 Transcription Factors - genetics SOXB1 Transcription Factors - metabolism Stromal Cells - cytology Stromal Cells - metabolism Transfection |
| title | Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome) |
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