Recent advances inex vivogene therapy for xeroderma pigmentosum patients
| Autor: | Sabine Scarzello, Thierry Magnaldo, Sophie Rouanet, Emilie Warrick, Yannick Gache |
|---|---|
| Rok vydání: | 2013 |
| Předmět: |
Genetics
congenital hereditary and neonatal diseases and abnormalities medicine.medical_specialty Xeroderma pigmentosum integumentary system business.industry Genetic enhancement Genodermatosis nutritional and metabolic diseases Cancer Dermatology Disease medicine.disease Bioinformatics Molecular genetics medicine Skin cancer skin and connective tissue diseases business Nucleotide excision repair |
| Zdroj: | Expert Review of Dermatology. 8:249-256 |
| ISSN: | 1746-9880 1746-9872 |
| Popis: | Perspectives of genetic treatment adapted to orphan monogenic genodermatosis have been fueled by tremendous progress in molecular genetics and gene transfer technologies. Xeroderma pigmentosum is a prototype of cancer prone syndromes; it is caused by severe alterations in nucleotide excision repair of DNA lesions introduced during exposure to sunlight. Here, the authors review the molecular basis of the disease and its impact on genotoxic insults caused by sunlight. The authors propose a compilation of preclinical results, revealing rational perspectives of cutaneous genetic correction of xeroderma pigmentosum with respect to appropriate specifications for this orphan and devastating disease. |
| Databáze: | OpenAIRE |
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