Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice
Attempts to treat congenital protein deficiencies using bone marrow-derived cells have been reported. These efforts have been based on the concepts of stem cell plasticity. However, it is considered more difficult to restore structural proteins than to restore secretory enzymes. This study aims to c...
Gespeichert in:
| Veröffentlicht in: | Proceedings of the National Academy of Sciences - PNAS 2010-08, Vol.107 (32), p.14345-14514 |
|---|---|
| Hauptverfasser: | , , , , , , , , , , , , , |
| Format: | Artikel |
| Sprache: | eng |
| Schlagworte: | |
| Online-Zugang: | Volltext |
| Tags: |
Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
|
| container_end_page | 14514 |
|---|---|
| container_issue | 32 |
| container_start_page | 14345 |
| container_title | Proceedings of the National Academy of Sciences - PNAS |
| container_volume | 107 |
| creator | Fujita, Yasuyuki Abe, Riichiro Inokuma, Daisuke Sasaki, Mikako Hoshina, Daichi Natsuga, Ken Nishie, Wataru McMillan, James R. Nakamura, Hideki Shimizu, Tadamichi Akiyama, Masashi Sawamura, Daisuke Shimizu, Hiroshi Lowy, Douglas |
| description | Attempts to treat congenital protein deficiencies using bone marrow-derived cells have been reported. These efforts have been based on the concepts of stem cell plasticity. However, it is considered more difficult to restore structural proteins than to restore secretory enzymes. This study aims to clarify whether bone marrow transplantation (BMT) treatment can rescue epidermolysis bullosa (EB) caused by defects in keratinocyte structural proteins. BMT treatment of adult collagen XVII (Col17) knockout mice induced donor-derived keratinocytes and Col17 expression associated with the recovery of hemidesmosomal structure and better skin manifestations, as well improving the survival rate. Both hematopoietic and mesenchymal stem cells have the potential to produce Col17 in the BMT treatment model. Furthermore, human cord blood CD34⁺ cells also differentiated into keratinocytes and expressed human skin component proteins in transplanted immunocompromised (NOD/SCID/γ null c ) mice. The current conventional BMT techniques have significant potential as a systemic therapeutic approach for the treatment of human EB. |
| doi_str_mv | 10.1073/pnas.1000044107 |
| format | Article |
| fullrecord | <record><control><sourceid>jstor_proqu</sourceid><recordid>TN_cdi_proquest_miscellaneous_748937471</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><jstor_id>25708904</jstor_id><sourcerecordid>25708904</sourcerecordid><originalsourceid>FETCH-LOGICAL-c532t-18b07cecb5df249c7288580d81e535e5083212aa2d81a594306138a6aba4b79b3</originalsourceid><addsrcrecordid>eNpdkU1v1DAYhC0EokvhzAlkcekprT9j-4IEVYFKlbjA2XKSdyGrxA52AvTGT-8b7dKl-GBb9jMjj4eQl5ydc2bkxRRDwR0OpfDgEdlw5nhVK8cekw1jwlRWCXVCnpWyQ8ppy56SE8HqmhllNuTP-xSBjiHn9IvOOcQyDSHOYe5TpBnKnHCiMPUd5DEMtAkFRogzHWFsEAc65TRDHyn8nhAtqy7EbtW2y1GahtvSF9osw5BKoGPqYKBj38Jz8mQbhgIvDusp-frh6svlp-rm88fry3c3VaulmCtuG2ZaaBvdbYVyrRHWYpbOctBSg2ZWCi5CEHgStFOS1VzaUIcmqMa4Rp6St3vfaWlG6FrMkMPgp9xj-FufQu8f3sT-u_-WfnrhhNA1Q4Ozg0FOPzDZ7Me-tDDgd0FaijfKOol_ypF88x-5S0uOmA4h6awxrkboYg-1OZWSYXv_FM782q1fu_XHblHx-t8E9_zfMhGgB2BVHu2Ml8JzJZVG5NUe2a3NHi20YdYxJe8AVQ-34A</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>743987796</pqid></control><display><type>article</type><title>Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice</title><source>Jstor Complete Legacy</source><source>MEDLINE</source><source>PubMed Central</source><source>Alma/SFX Local Collection</source><source>Free Full-Text Journals in Chemistry</source><creator>Fujita, Yasuyuki ; Abe, Riichiro ; Inokuma, Daisuke ; Sasaki, Mikako ; Hoshina, Daichi ; Natsuga, Ken ; Nishie, Wataru ; McMillan, James R. ; Nakamura, Hideki ; Shimizu, Tadamichi ; Akiyama, Masashi ; Sawamura, Daisuke ; Shimizu, Hiroshi ; Lowy, Douglas</creator><creatorcontrib>Fujita, Yasuyuki ; Abe, Riichiro ; Inokuma, Daisuke ; Sasaki, Mikako ; Hoshina, Daichi ; Natsuga, Ken ; Nishie, Wataru ; McMillan, James R. ; Nakamura, Hideki ; Shimizu, Tadamichi ; Akiyama, Masashi ; Sawamura, Daisuke ; Shimizu, Hiroshi ; Lowy, Douglas</creatorcontrib><description>Attempts to treat congenital protein deficiencies using bone marrow-derived cells have been reported. These efforts have been based on the concepts of stem cell plasticity. However, it is considered more difficult to restore structural proteins than to restore secretory enzymes. This study aims to clarify whether bone marrow transplantation (BMT) treatment can rescue epidermolysis bullosa (EB) caused by defects in keratinocyte structural proteins. BMT treatment of adult collagen XVII (Col17) knockout mice induced donor-derived keratinocytes and Col17 expression associated with the recovery of hemidesmosomal structure and better skin manifestations, as well improving the survival rate. Both hematopoietic and mesenchymal stem cells have the potential to produce Col17 in the BMT treatment model. Furthermore, human cord blood CD34⁺ cells also differentiated into keratinocytes and expressed human skin component proteins in transplanted immunocompromised (NOD/SCID/γ null c ) mice. The current conventional BMT techniques have significant potential as a systemic therapeutic approach for the treatment of human EB.</description><identifier>ISSN: 0027-8424</identifier><identifier>EISSN: 1091-6490</identifier><identifier>DOI: 10.1073/pnas.1000044107</identifier><identifier>PMID: 20660747</identifier><language>eng</language><publisher>United States: National Academy of Sciences</publisher><subject>Animals ; Autoantigens - biosynthesis ; Basement Membrane - chemistry ; Basement Membrane - metabolism ; Biological Sciences ; Blood cells ; Bone marrow ; Bone Marrow Transplantation - physiology ; Cell transplantation ; Collagen Type XVII ; Epidermal cells ; Epidermis ; Epidermolysis bullosa ; Epidermolysis Bullosa - therapy ; Humans ; Keratinocytes ; Keratinocytes - cytology ; Medical treatment ; Membrane Proteins - biosynthesis ; Mesenchymal stem cells ; Messenger RNA ; Mice ; Mice, SCID ; Non-Fibrillar Collagens - biosynthesis ; Non-Fibrillar Collagens - deficiency ; Proteins ; Rodents ; Skin ; Stem Cell Transplantation ; Stem cells ; Survival Rate ; Transplants & implants ; Treatment Outcome</subject><ispartof>Proceedings of the National Academy of Sciences - PNAS, 2010-08, Vol.107 (32), p.14345-14514</ispartof><rights>Copyright National Academy of Sciences Aug 10, 2010</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c532t-18b07cecb5df249c7288580d81e535e5083212aa2d81a594306138a6aba4b79b3</citedby><cites>FETCH-LOGICAL-c532t-18b07cecb5df249c7288580d81e535e5083212aa2d81a594306138a6aba4b79b3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Uhttp://www.pnas.org/content/107/32.cover.gif</thumbnail><linktopdf>$$Uhttps://www.jstor.org/stable/pdf/25708904$$EPDF$$P50$$Gjstor$$H</linktopdf><linktohtml>$$Uhttps://www.jstor.org/stable/25708904$$EHTML$$P50$$Gjstor$$H</linktohtml><link.rule.ids>230,314,723,776,780,799,881,27901,27902,53766,53768,57992,58225</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/20660747$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Fujita, Yasuyuki</creatorcontrib><creatorcontrib>Abe, Riichiro</creatorcontrib><creatorcontrib>Inokuma, Daisuke</creatorcontrib><creatorcontrib>Sasaki, Mikako</creatorcontrib><creatorcontrib>Hoshina, Daichi</creatorcontrib><creatorcontrib>Natsuga, Ken</creatorcontrib><creatorcontrib>Nishie, Wataru</creatorcontrib><creatorcontrib>McMillan, James R.</creatorcontrib><creatorcontrib>Nakamura, Hideki</creatorcontrib><creatorcontrib>Shimizu, Tadamichi</creatorcontrib><creatorcontrib>Akiyama, Masashi</creatorcontrib><creatorcontrib>Sawamura, Daisuke</creatorcontrib><creatorcontrib>Shimizu, Hiroshi</creatorcontrib><creatorcontrib>Lowy, Douglas</creatorcontrib><title>Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice</title><title>Proceedings of the National Academy of Sciences - PNAS</title><addtitle>Proc Natl Acad Sci U S A</addtitle><description>Attempts to treat congenital protein deficiencies using bone marrow-derived cells have been reported. These efforts have been based on the concepts of stem cell plasticity. However, it is considered more difficult to restore structural proteins than to restore secretory enzymes. This study aims to clarify whether bone marrow transplantation (BMT) treatment can rescue epidermolysis bullosa (EB) caused by defects in keratinocyte structural proteins. BMT treatment of adult collagen XVII (Col17) knockout mice induced donor-derived keratinocytes and Col17 expression associated with the recovery of hemidesmosomal structure and better skin manifestations, as well improving the survival rate. Both hematopoietic and mesenchymal stem cells have the potential to produce Col17 in the BMT treatment model. Furthermore, human cord blood CD34⁺ cells also differentiated into keratinocytes and expressed human skin component proteins in transplanted immunocompromised (NOD/SCID/γ null c ) mice. The current conventional BMT techniques have significant potential as a systemic therapeutic approach for the treatment of human EB.</description><subject>Animals</subject><subject>Autoantigens - biosynthesis</subject><subject>Basement Membrane - chemistry</subject><subject>Basement Membrane - metabolism</subject><subject>Biological Sciences</subject><subject>Blood cells</subject><subject>Bone marrow</subject><subject>Bone Marrow Transplantation - physiology</subject><subject>Cell transplantation</subject><subject>Collagen Type XVII</subject><subject>Epidermal cells</subject><subject>Epidermis</subject><subject>Epidermolysis bullosa</subject><subject>Epidermolysis Bullosa - therapy</subject><subject>Humans</subject><subject>Keratinocytes</subject><subject>Keratinocytes - cytology</subject><subject>Medical treatment</subject><subject>Membrane Proteins - biosynthesis</subject><subject>Mesenchymal stem cells</subject><subject>Messenger RNA</subject><subject>Mice</subject><subject>Mice, SCID</subject><subject>Non-Fibrillar Collagens - biosynthesis</subject><subject>Non-Fibrillar Collagens - deficiency</subject><subject>Proteins</subject><subject>Rodents</subject><subject>Skin</subject><subject>Stem Cell Transplantation</subject><subject>Stem cells</subject><subject>Survival Rate</subject><subject>Transplants & implants</subject><subject>Treatment Outcome</subject><issn>0027-8424</issn><issn>1091-6490</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2010</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNpdkU1v1DAYhC0EokvhzAlkcekprT9j-4IEVYFKlbjA2XKSdyGrxA52AvTGT-8b7dKl-GBb9jMjj4eQl5ydc2bkxRRDwR0OpfDgEdlw5nhVK8cekw1jwlRWCXVCnpWyQ8ppy56SE8HqmhllNuTP-xSBjiHn9IvOOcQyDSHOYe5TpBnKnHCiMPUd5DEMtAkFRogzHWFsEAc65TRDHyn8nhAtqy7EbtW2y1GahtvSF9osw5BKoGPqYKBj38Jz8mQbhgIvDusp-frh6svlp-rm88fry3c3VaulmCtuG2ZaaBvdbYVyrRHWYpbOctBSg2ZWCi5CEHgStFOS1VzaUIcmqMa4Rp6St3vfaWlG6FrMkMPgp9xj-FufQu8f3sT-u_-WfnrhhNA1Q4Ozg0FOPzDZ7Me-tDDgd0FaijfKOol_ypF88x-5S0uOmA4h6awxrkboYg-1OZWSYXv_FM782q1fu_XHblHx-t8E9_zfMhGgB2BVHu2Ml8JzJZVG5NUe2a3NHi20YdYxJe8AVQ-34A</recordid><startdate>20100810</startdate><enddate>20100810</enddate><creator>Fujita, Yasuyuki</creator><creator>Abe, Riichiro</creator><creator>Inokuma, Daisuke</creator><creator>Sasaki, Mikako</creator><creator>Hoshina, Daichi</creator><creator>Natsuga, Ken</creator><creator>Nishie, Wataru</creator><creator>McMillan, James R.</creator><creator>Nakamura, Hideki</creator><creator>Shimizu, Tadamichi</creator><creator>Akiyama, Masashi</creator><creator>Sawamura, Daisuke</creator><creator>Shimizu, Hiroshi</creator><creator>Lowy, Douglas</creator><general>National Academy of Sciences</general><general>National Acad Sciences</general><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>7QG</scope><scope>7QL</scope><scope>7QP</scope><scope>7QR</scope><scope>7SN</scope><scope>7SS</scope><scope>7T5</scope><scope>7TK</scope><scope>7TM</scope><scope>7TO</scope><scope>7U9</scope><scope>8FD</scope><scope>C1K</scope><scope>FR3</scope><scope>H94</scope><scope>M7N</scope><scope>P64</scope><scope>RC3</scope><scope>7X8</scope><scope>5PM</scope></search><sort><creationdate>20100810</creationdate><title>Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice</title><author>Fujita, Yasuyuki ; Abe, Riichiro ; Inokuma, Daisuke ; Sasaki, Mikako ; Hoshina, Daichi ; Natsuga, Ken ; Nishie, Wataru ; McMillan, James R. ; Nakamura, Hideki ; Shimizu, Tadamichi ; Akiyama, Masashi ; Sawamura, Daisuke ; Shimizu, Hiroshi ; Lowy, Douglas</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c532t-18b07cecb5df249c7288580d81e535e5083212aa2d81a594306138a6aba4b79b3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2010</creationdate><topic>Animals</topic><topic>Autoantigens - biosynthesis</topic><topic>Basement Membrane - chemistry</topic><topic>Basement Membrane - metabolism</topic><topic>Biological Sciences</topic><topic>Blood cells</topic><topic>Bone marrow</topic><topic>Bone Marrow Transplantation - physiology</topic><topic>Cell transplantation</topic><topic>Collagen Type XVII</topic><topic>Epidermal cells</topic><topic>Epidermis</topic><topic>Epidermolysis bullosa</topic><topic>Epidermolysis Bullosa - therapy</topic><topic>Humans</topic><topic>Keratinocytes</topic><topic>Keratinocytes - cytology</topic><topic>Medical treatment</topic><topic>Membrane Proteins - biosynthesis</topic><topic>Mesenchymal stem cells</topic><topic>Messenger RNA</topic><topic>Mice</topic><topic>Mice, SCID</topic><topic>Non-Fibrillar Collagens - biosynthesis</topic><topic>Non-Fibrillar Collagens - deficiency</topic><topic>Proteins</topic><topic>Rodents</topic><topic>Skin</topic><topic>Stem Cell Transplantation</topic><topic>Stem cells</topic><topic>Survival Rate</topic><topic>Transplants & implants</topic><topic>Treatment Outcome</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Fujita, Yasuyuki</creatorcontrib><creatorcontrib>Abe, Riichiro</creatorcontrib><creatorcontrib>Inokuma, Daisuke</creatorcontrib><creatorcontrib>Sasaki, Mikako</creatorcontrib><creatorcontrib>Hoshina, Daichi</creatorcontrib><creatorcontrib>Natsuga, Ken</creatorcontrib><creatorcontrib>Nishie, Wataru</creatorcontrib><creatorcontrib>McMillan, James R.</creatorcontrib><creatorcontrib>Nakamura, Hideki</creatorcontrib><creatorcontrib>Shimizu, Tadamichi</creatorcontrib><creatorcontrib>Akiyama, Masashi</creatorcontrib><creatorcontrib>Sawamura, Daisuke</creatorcontrib><creatorcontrib>Shimizu, Hiroshi</creatorcontrib><creatorcontrib>Lowy, Douglas</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Animal Behavior Abstracts</collection><collection>Bacteriology Abstracts (Microbiology B)</collection><collection>Calcium & Calcified Tissue Abstracts</collection><collection>Chemoreception Abstracts</collection><collection>Ecology Abstracts</collection><collection>Entomology Abstracts (Full archive)</collection><collection>Immunology Abstracts</collection><collection>Neurosciences Abstracts</collection><collection>Nucleic Acids Abstracts</collection><collection>Oncogenes and Growth Factors Abstracts</collection><collection>Virology and AIDS Abstracts</collection><collection>Technology Research Database</collection><collection>Environmental Sciences and Pollution Management</collection><collection>Engineering Research Database</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>Algology Mycology and Protozoology Abstracts (Microbiology C)</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Proceedings of the National Academy of Sciences - PNAS</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Fujita, Yasuyuki</au><au>Abe, Riichiro</au><au>Inokuma, Daisuke</au><au>Sasaki, Mikako</au><au>Hoshina, Daichi</au><au>Natsuga, Ken</au><au>Nishie, Wataru</au><au>McMillan, James R.</au><au>Nakamura, Hideki</au><au>Shimizu, Tadamichi</au><au>Akiyama, Masashi</au><au>Sawamura, Daisuke</au><au>Shimizu, Hiroshi</au><au>Lowy, Douglas</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice</atitle><jtitle>Proceedings of the National Academy of Sciences - PNAS</jtitle><addtitle>Proc Natl Acad Sci U S A</addtitle><date>2010-08-10</date><risdate>2010</risdate><volume>107</volume><issue>32</issue><spage>14345</spage><epage>14514</epage><pages>14345-14514</pages><issn>0027-8424</issn><eissn>1091-6490</eissn><abstract>Attempts to treat congenital protein deficiencies using bone marrow-derived cells have been reported. These efforts have been based on the concepts of stem cell plasticity. However, it is considered more difficult to restore structural proteins than to restore secretory enzymes. This study aims to clarify whether bone marrow transplantation (BMT) treatment can rescue epidermolysis bullosa (EB) caused by defects in keratinocyte structural proteins. BMT treatment of adult collagen XVII (Col17) knockout mice induced donor-derived keratinocytes and Col17 expression associated with the recovery of hemidesmosomal structure and better skin manifestations, as well improving the survival rate. Both hematopoietic and mesenchymal stem cells have the potential to produce Col17 in the BMT treatment model. Furthermore, human cord blood CD34⁺ cells also differentiated into keratinocytes and expressed human skin component proteins in transplanted immunocompromised (NOD/SCID/γ null c ) mice. The current conventional BMT techniques have significant potential as a systemic therapeutic approach for the treatment of human EB.</abstract><cop>United States</cop><pub>National Academy of Sciences</pub><pmid>20660747</pmid><doi>10.1073/pnas.1000044107</doi><tpages>170</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0027-8424 |
| ispartof | Proceedings of the National Academy of Sciences - PNAS, 2010-08, Vol.107 (32), p.14345-14514 |
| issn | 0027-8424 1091-6490 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_748937471 |
| source | Jstor Complete Legacy; MEDLINE; PubMed Central; Alma/SFX Local Collection; Free Full-Text Journals in Chemistry |
| subjects | Animals Autoantigens - biosynthesis Basement Membrane - chemistry Basement Membrane - metabolism Biological Sciences Blood cells Bone marrow Bone Marrow Transplantation - physiology Cell transplantation Collagen Type XVII Epidermal cells Epidermis Epidermolysis bullosa Epidermolysis Bullosa - therapy Humans Keratinocytes Keratinocytes - cytology Medical treatment Membrane Proteins - biosynthesis Mesenchymal stem cells Messenger RNA Mice Mice, SCID Non-Fibrillar Collagens - biosynthesis Non-Fibrillar Collagens - deficiency Proteins Rodents Skin Stem Cell Transplantation Stem cells Survival Rate Transplants & implants Treatment Outcome |
| title | Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-02-01T15%3A06%3A53IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-jstor_proqu&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Bone%20marrow%20transplantation%20restores%20epidermal%20basement%20membrane%20protein%20expression%20and%20rescues%20epidermolysis%20bullosa%20model%20mice&rft.jtitle=Proceedings%20of%20the%20National%20Academy%20of%20Sciences%20-%20PNAS&rft.au=Fujita,%20Yasuyuki&rft.date=2010-08-10&rft.volume=107&rft.issue=32&rft.spage=14345&rft.epage=14514&rft.pages=14345-14514&rft.issn=0027-8424&rft.eissn=1091-6490&rft_id=info:doi/10.1073/pnas.1000044107&rft_dat=%3Cjstor_proqu%3E25708904%3C/jstor_proqu%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=743987796&rft_id=info:pmid/20660747&rft_jstor_id=25708904&rfr_iscdi=true |