Preclinical Corrective Gene Transfer in Xeroderma Pigmentosum Human Skin Stem Cells
Xeroderma pigmentosum (XP) is a devastating disease associated with dramatic skin cancer proneness. XP cells are deficient in nucleotide excision repair (NER) of bulky DNA adducts including ultraviolet (UV)-induced mutagenic lesions. Approaches of corrective gene transfer in NER-deficient keratinocy...
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| Veröffentlicht in: | Molecular therapy 2012-04, Vol.20 (4), p.798-807 |
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| creator | Warrick, Emilie Garcia, Marta Chagnoleau, Corinne Chevallier, Odile Bergoglio, Valérie Sartori, Daniela Mavilio, Fulvio Angulo, Jaime F Avril, Marie-Françoise Sarasin, Alain Larcher, Fernando Del Rio, Marcela Bernerd, Françoise Magnaldo, Thierry |
| description | Xeroderma pigmentosum (XP) is a devastating disease associated with dramatic skin cancer proneness. XP cells are deficient in nucleotide excision repair (NER) of bulky DNA adducts including ultraviolet (UV)-induced mutagenic lesions. Approaches of corrective gene transfer in NER-deficient keratinocyte stem cells hold great hope for the long-term treatment of XP patients. To face this challenge, we developed a retrovirus-based strategy to safely transduce the wild-type XPC gene into clonogenic human primary XP-C keratinocytes. De novo expression of XPC was maintained in both mass population and derived independent candidate stem cells (holoclones) after more than 130 population doublings (PD) in culture upon serial propagation (>1040 cells). Analyses of retrovirus integration sequences in isolated keratinocyte stem cells suggested the absence of adverse effects such as oncogenic activation or clonal expansion. Furthermore, corrected XP-C keratinocytes exhibited full NER capacity as well as normal features of epidermal differentiation in both organotypic skin cultures and in a preclinical murine model of human skin regeneration in vivo. The achievement of a long-term genetic correction of XP-C epidermal stem cells constitutes the first preclinical model of ex vivo gene therapy for XP-C patients. |
| doi_str_mv | 10.1038/mt.2011.233 |
| format | Article |
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XP cells are deficient in nucleotide excision repair (NER) of bulky DNA adducts including ultraviolet (UV)-induced mutagenic lesions. Approaches of corrective gene transfer in NER-deficient keratinocyte stem cells hold great hope for the long-term treatment of XP patients. To face this challenge, we developed a retrovirus-based strategy to safely transduce the wild-type XPC gene into clonogenic human primary XP-C keratinocytes. De novo expression of XPC was maintained in both mass population and derived independent candidate stem cells (holoclones) after more than 130 population doublings (PD) in culture upon serial propagation (>1040 cells). Analyses of retrovirus integration sequences in isolated keratinocyte stem cells suggested the absence of adverse effects such as oncogenic activation or clonal expansion. Furthermore, corrected XP-C keratinocytes exhibited full NER capacity as well as normal features of epidermal differentiation in both organotypic skin cultures and in a preclinical murine model of human skin regeneration in vivo. The achievement of a long-term genetic correction of XP-C epidermal stem cells constitutes the first preclinical model of ex vivo gene therapy for XP-C patients.</description><identifier>ISSN: 1525-0016</identifier><identifier>EISSN: 1525-0024</identifier><identifier>DOI: 10.1038/mt.2011.233</identifier><identifier>PMID: 22068429</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Blotting, Southern ; Blotting, Western ; Cells, Cultured ; Disease ; DNA damage ; DNA repair ; DNA-Binding Proteins - genetics ; DNA-Binding Proteins - metabolism ; Epidermis - cytology ; Epidermis - metabolism ; Flow Cytometry ; Gene therapy ; Genetic Therapy ; Genomes ; Humans ; Keratinocytes - cytology ; Keratinocytes - metabolism ; Life Sciences ; Mutagenesis ; Original ; Real-Time Polymerase Chain Reaction ; RNA polymerase ; Skin - cytology ; Skin - metabolism ; Skin cancer ; Stem cells ; Stem Cells - cytology ; Stem Cells - metabolism ; Tumors ; Xeroderma Pigmentosum - metabolism ; Xeroderma Pigmentosum - therapy</subject><ispartof>Molecular therapy, 2012-04, Vol.20 (4), p.798-807</ispartof><rights>2012 The American Society of Gene & Cell Therapy</rights><rights>Copyright Nature Publishing Group Apr 2012</rights><rights>Attribution - NonCommercial - NoDerivatives</rights><rights>Copyright © 2012 The American Society of Gene & Cell Therapy 2012 The American Society of Gene & Cell Therapy</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c521t-b242bdffc68d3249428488e399595d68de42db28a5c8630604a20fc9b29941683</citedby><cites>FETCH-LOGICAL-c521t-b242bdffc68d3249428488e399595d68de42db28a5c8630604a20fc9b29941683</cites><orcidid>0000-0003-2773-3178 ; 0000-0002-2445-6016 ; 0000-0003-0459-4320</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/PMC3321585/pdf/$$EPDF$$P50$$Gpubmedcentral$$H</linktopdf><linktohtml>$$Uhttps://www.proquest.com/docview/1792073197?pq-origsite=primo$$EHTML$$P50$$Gproquest$$H</linktohtml><link.rule.ids>230,315,728,781,785,886,27929,27930,53796,53798,64390,64392,64394,72474</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/22068429$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink><backlink>$$Uhttps://hal.science/hal-02325743$$DView record in HAL$$Hfree_for_read</backlink></links><search><creatorcontrib>Warrick, Emilie</creatorcontrib><creatorcontrib>Garcia, Marta</creatorcontrib><creatorcontrib>Chagnoleau, Corinne</creatorcontrib><creatorcontrib>Chevallier, Odile</creatorcontrib><creatorcontrib>Bergoglio, Valérie</creatorcontrib><creatorcontrib>Sartori, Daniela</creatorcontrib><creatorcontrib>Mavilio, Fulvio</creatorcontrib><creatorcontrib>Angulo, Jaime F</creatorcontrib><creatorcontrib>Avril, Marie-Françoise</creatorcontrib><creatorcontrib>Sarasin, Alain</creatorcontrib><creatorcontrib>Larcher, Fernando</creatorcontrib><creatorcontrib>Del Rio, Marcela</creatorcontrib><creatorcontrib>Bernerd, Françoise</creatorcontrib><creatorcontrib>Magnaldo, Thierry</creatorcontrib><title>Preclinical Corrective Gene Transfer in Xeroderma Pigmentosum Human Skin Stem Cells</title><title>Molecular therapy</title><addtitle>Mol Ther</addtitle><description>Xeroderma pigmentosum (XP) is a devastating disease associated with dramatic skin cancer proneness. XP cells are deficient in nucleotide excision repair (NER) of bulky DNA adducts including ultraviolet (UV)-induced mutagenic lesions. Approaches of corrective gene transfer in NER-deficient keratinocyte stem cells hold great hope for the long-term treatment of XP patients. To face this challenge, we developed a retrovirus-based strategy to safely transduce the wild-type XPC gene into clonogenic human primary XP-C keratinocytes. De novo expression of XPC was maintained in both mass population and derived independent candidate stem cells (holoclones) after more than 130 population doublings (PD) in culture upon serial propagation (>1040 cells). Analyses of retrovirus integration sequences in isolated keratinocyte stem cells suggested the absence of adverse effects such as oncogenic activation or clonal expansion. Furthermore, corrected XP-C keratinocytes exhibited full NER capacity as well as normal features of epidermal differentiation in both organotypic skin cultures and in a preclinical murine model of human skin regeneration in vivo. The achievement of a long-term genetic correction of XP-C epidermal stem cells constitutes the first preclinical model of ex vivo gene therapy for XP-C patients.</description><subject>Blotting, Southern</subject><subject>Blotting, Western</subject><subject>Cells, Cultured</subject><subject>Disease</subject><subject>DNA damage</subject><subject>DNA repair</subject><subject>DNA-Binding Proteins - genetics</subject><subject>DNA-Binding Proteins - metabolism</subject><subject>Epidermis - cytology</subject><subject>Epidermis - metabolism</subject><subject>Flow Cytometry</subject><subject>Gene therapy</subject><subject>Genetic Therapy</subject><subject>Genomes</subject><subject>Humans</subject><subject>Keratinocytes - cytology</subject><subject>Keratinocytes - metabolism</subject><subject>Life Sciences</subject><subject>Mutagenesis</subject><subject>Original</subject><subject>Real-Time Polymerase Chain Reaction</subject><subject>RNA polymerase</subject><subject>Skin - cytology</subject><subject>Skin - metabolism</subject><subject>Skin cancer</subject><subject>Stem cells</subject><subject>Stem Cells - cytology</subject><subject>Stem Cells - metabolism</subject><subject>Tumors</subject><subject>Xeroderma Pigmentosum - metabolism</subject><subject>Xeroderma Pigmentosum - 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XP cells are deficient in nucleotide excision repair (NER) of bulky DNA adducts including ultraviolet (UV)-induced mutagenic lesions. Approaches of corrective gene transfer in NER-deficient keratinocyte stem cells hold great hope for the long-term treatment of XP patients. To face this challenge, we developed a retrovirus-based strategy to safely transduce the wild-type XPC gene into clonogenic human primary XP-C keratinocytes. De novo expression of XPC was maintained in both mass population and derived independent candidate stem cells (holoclones) after more than 130 population doublings (PD) in culture upon serial propagation (>1040 cells). Analyses of retrovirus integration sequences in isolated keratinocyte stem cells suggested the absence of adverse effects such as oncogenic activation or clonal expansion. Furthermore, corrected XP-C keratinocytes exhibited full NER capacity as well as normal features of epidermal differentiation in both organotypic skin cultures and in a preclinical murine model of human skin regeneration in vivo. The achievement of a long-term genetic correction of XP-C epidermal stem cells constitutes the first preclinical model of ex vivo gene therapy for XP-C patients.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>22068429</pmid><doi>10.1038/mt.2011.233</doi><tpages>10</tpages><orcidid>https://orcid.org/0000-0003-2773-3178</orcidid><orcidid>https://orcid.org/0000-0002-2445-6016</orcidid><orcidid>https://orcid.org/0000-0003-0459-4320</orcidid><oa>free_for_read</oa></addata></record> |
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| subjects | Blotting, Southern Blotting, Western Cells, Cultured Disease DNA damage DNA repair DNA-Binding Proteins - genetics DNA-Binding Proteins - metabolism Epidermis - cytology Epidermis - metabolism Flow Cytometry Gene therapy Genetic Therapy Genomes Humans Keratinocytes - cytology Keratinocytes - metabolism Life Sciences Mutagenesis Original Real-Time Polymerase Chain Reaction RNA polymerase Skin - cytology Skin - metabolism Skin cancer Stem cells Stem Cells - cytology Stem Cells - metabolism Tumors Xeroderma Pigmentosum - metabolism Xeroderma Pigmentosum - therapy |
| title | Preclinical Corrective Gene Transfer in Xeroderma Pigmentosum Human Skin Stem Cells |
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