ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Efficient KRT14 targeting and functional characterization of transplanted human keratinocytes for the treatment of epidermolysis bullosa simplex.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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Who cites it
22 citing papers in PubMed, 46 citations in OpenAlex.
- Treatment Landscape for Epidermolysis Bullosa Simplex: A Review of Established and Emerging Therapies.Pediatric dermatology · 2026Review
- An anoikis-based gene signature for predicting prognosis in malignant pleural mesothelioma and revealing immune infiltration.Journal of cancer research and clinical oncology · 2023Article
- Differential gene screening and bioinformatics analysis of epidermal stem cells and dermal fibroblasts during skin aging.Scientific reports · 2022Article
- Protective effect of bone marrow mesenchymal stem cells modified with klotho on renal ischemia-reperfusion injury.Renal failure · 2019Article
- Cut and Paste: Efficient Homology-Directed Repair of a Dominant Negative KRT14 Mutation via CRISPR/Cas9 Nickases.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes.Molecular therapy. Nucleic acids · 2016Article
- Robust Lentiviral Gene Delivery But Limited Transduction Capacity of Commonly Used Adeno-Associated Viral Serotypes in Xenotransplanted Human Skin.Human gene therapy methods · 2015Article
- Adeno-associated Virus as a Mammalian DNA Vector.Microbiology spectrum · 2015Review
- Tropism-modified AAV vectors overcome barriers to successful cutaneous therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Gene therapy for skin diseases.Cold Spring Harbor perspectives in medicine · 2014Review
- Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Repairing without cutting: a safer alternative to gene correction?Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Selection and evaluation of clinically relevant AAV variants in a xenograft liver model.Nature · 2014Article
- Local delivery of gene-modifying triplex-forming molecules to the epidermis.The Journal of investigative dermatology · 2013Article
- In vitro analysis of photosensitizer accumulation for assessment of applicability of fluorescence diagnosis of squamous cell carcinoma of epidermolysis bullosa patients.BioMed research international · 2013Article
- Progress towards genetic and pharmacological therapies for keratin genodermatoses: current perspective and future promise.Experimental dermatology · 2012Review
- Defining keratin protein function in skin epithelia: epidermolysis bullosa simplex and its aftermath.The Journal of investigative dermatology · 2012Review
- Normal collagen and bone production by gene-targeted human osteogenesis imperfecta iPSCs.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Asymmetric bidirectional transcription from the FSHD-causing D4Z4 array modulates DUX4 production.PloS one · 2012Article
- AAV-mediated gene targeting methods for human cells.Nature protocols · 2011Article
Corrections and comments
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
Inherited skin blistering conditions collectively named epidermolysis bullosa (EB) cause significant morbidity and mortality due to the compromise of the skin's barrier function, the pain of blisters, inflammation, and in some cases scaring and cancer. The simplex form of EB is usually caused by dominantly inherited mutations in KRT5 or KRT14. These mutations result in the production of proteins with dominant-negative activity that disrupt polymerization of intermediate filaments in the basal keratinocyte layer and result in a weak epidermal-dermal junction. The genome of adeno-associated virus (AAV) vectors can recombine with chromosomal sequence so that mutations can be corrected, or production of proteins with dominant-negative activity can be disrupted. We demonstrate a clinically feasible strategy for efficient targeting of the KRT14 gene in normal and EB-affected human keratinocytes. Using a gene-targeting vector with promoter trap design, targeted alteration of one allele of KRT14 occurred in 100% of transduced cells and transduction frequencies ranged from 0.1 to 0.6% of total cells. EBS patient keratinocytes with precise modifications of the mutant allele are preferentially recovered from targeted cell populations. Single epidermal stem cell clones produced histologically normal skin grafts after transplantation to athymic mice and could generate a sufficient number of cells to transplant the entire skin surface of an individual.
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