دورية أكاديمية
Genetic Correction of Stem Cells in the Treatment of Inherited Diseases and Focus on Xeroderma Pigmentosum
| العنوان: | Genetic Correction of Stem Cells in the Treatment of Inherited Diseases and Focus on Xeroderma Pigmentosum |
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| المؤلفون: | Rouanet, Sophie, Warrick, Emilie, Gache, Yannick, Scarzello, Sabine, Avril, Marie-Françoise, Bernerd, Françoise, Magnaldo, Thierry |
| المساهمون: | Institut de Recherche sur le Cancer et le Vieillissement (IRCAN), Université Nice Sophia Antipolis (1965 - 2019) (UNS)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Côte d'Azur (UniCA), L'Oréal Recherche France (L'Oréal Recherche), L'OREAL, Hôpital Cochin AP-HP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Université Pierre et Marie Curie - Paris 6 (UPMC) |
| المصدر: | ISSN: 1661-6596. |
| بيانات النشر: | HAL CCSD MDPI |
| سنة النشر: | 2013 |
| المجموعة: | HAL Université Côte d'Azur |
| مصطلحات موضوعية: | [SDV.BBM]Life Sciences [q-bio]/Biochemistry, Molecular Biology |
| الوصف: | International audience ; Somatic stem cells ensure tissue renewal along life and healing of injuries. Their safe isolation, genetic manipulation ex vivo and reinfusion in patients suffering from life threatening immune deficiencies (for example, severe combined immunodeficiency (SCID)) have demonstrated the efficacy of ex vivo gene therapy. Similarly, adult epidermal stem cells have the capacity to renew epidermis, the fully differentiated, protective envelope of our body. Stable skin replacement of severely burned patients have proven life saving. Xeroderma pigmentosum (XP) is a devastating disease due to severe defects in the repair of mutagenic DNA lesions introduced upon exposure to solar radiations. Most patients die from the consequences of budding hundreds of skin cancers in the absence of photoprotection. We have developed a safe procedure of genetic correction of epidermal stem cells isolated from XP patients. Preclinical and safety assessments indicate successful correction of XP epidermal stem cells in the long term and their capacity to regenerate a normal skin with full capacities of DNA repair. |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| العلاقة: | hal-01583886; https://hal.sorbonne-universite.fr/hal-01583886Test; https://hal.sorbonne-universite.fr/hal-01583886/documentTest; https://hal.sorbonne-universite.fr/hal-01583886/file/ijms-14-20019.pdfTest |
| DOI: | 10.3390/ijms141020019 |
| الإتاحة: | https://doi.org/10.3390/ijms141020019Test https://hal.sorbonne-universite.fr/hal-01583886Test https://hal.sorbonne-universite.fr/hal-01583886/documentTest https://hal.sorbonne-universite.fr/hal-01583886/file/ijms-14-20019.pdfTest |
| حقوق: | http://creativecommons.org/licenses/byTest/ ; info:eu-repo/semantics/OpenAccess |
| رقم الانضمام: | edsbas.F99BD782 |
| قاعدة البيانات: | BASE |
| DOI: | 10.3390/ijms141020019 |
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