ArticleMolecular therapy : the journal of the American Society of Gene Therapy2012
Preclinical corrective gene transfer in xeroderma pigmentosum human skin stem cells.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 58 citations in OpenAlex.
- Type I interferon signaling defines a novel disease signature in xeroderma pigmentosum C human keratinocytes.Scientific reports · 2026Article
- Development of pathological skin models: from conventional techniques to 3D bioprinting.Frontiers in bioengineering and biotechnology · 2025Review
- Generation and characterization of CRISPR-Cas9-mediated XPC gene knockout in human skin cells.Scientific reports · 2024Article
- Article
- Xeroderma Pigmentosum: General Aspects and Management.Journal of personalized medicine · 2021Review
- CRISPR-Pass: Gene Rescue of Nonsense Mutations Using Adenine Base Editors.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Review
- Review
- Ultraviolet Radiation-Induced Skin Aging: The Role of DNA Damage and Oxidative Stress in Epidermal Stem Cell Damage Mediated Skin Aging.Stem cells international · 2016Review
- A single epidermal stem cell strategy for safe ex vivo gene therapy.EMBO molecular medicine · 2015Article
- Oxidative and energy metabolism as potential clues for clinical heterogeneity in nucleotide excision repair disorders.The Journal of investigative dermatology · 2015Review
- Gene therapy for skin diseases.Cold Spring Harbor perspectives in medicine · 2014Review
- Genetic correction of stem cells in the treatment of inherited diseases and focus on xeroderma pigmentosum.International journal of molecular sciences · 2013Review
- Article
- Xeroderma pigmentosum and its dental implications.European journal of dentistryArticle
- Current Therapeutic Strategies of Xeroderma Pigmentosum.Indian journal of dermatologyReview
- Gene Therapy and its Application in Dermatology.Indian journal of dermatologyReview
Corrections and comments
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Authors and funding
14 authors at 9 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
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 (>10(40) 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.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.