ArticleProceedings of the National Academy of Sciences of the United States of America2001
In vivo restoration of laminin 5 beta 3 expression and function in junctional epidermolysis bullosa.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2001. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
36 citing papers in PubMed, 126 citations in OpenAlex.
- Gentamicin Induces Laminin 332 and Improves Wound Healing in Junctional Epidermolysis Bullosa Patients with Nonsense Mutations.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Trial
- Tapping basement membrane motifs: Oral junctional epithelium for surface-mediated soft tissue attachment to prevent failure of percutaneous devices.Acta biomaterialia · 2022Article
- Discovery and validation of human genomic safe harbor sites for gene and cell therapies.Cell reports methods · 2022Article
- Leading edge: emerging drug, cell, and gene therapies for junctional epidermolysis bullosa.Expert opinion on biological therapy · 2020Review
- Microprocessor-dependent processing of splice site overlapping microRNA exons does not result in changes in alternative splicing.RNA (New York, N.Y.) · 2018Article
- Gentamicin inducesProceedings of the National Academy of Sciences of the United States of America · 2018Article
- Immunology of Wound Healing.Current dermatology reports · 2018Review
- Gene editing toward the use of autologous therapies in recessive dystrophic epidermolysis bullosa.Translational research : the journal of laboratory and clinical medicine · 2016Review
- Stem Cells in Skin Regeneration, Wound Healing, and Their Clinical Applications.International journal of molecular sciences · 2015Review
- Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Dermatology: Where are We Coming from and Where are We Going to?Frontiers in medicine · 2014Article
- Keratinocytes from induced pluripotent stem cells in junctional epidermolysis bullosa.The Journal of investigative dermatology · 2013Article
- Nucleus pulposus cell-matrix interactions with laminins.European cells & materials · 2011Article
- CYLD inhibits tumorigenesis and metastasis by blocking JNK/AP1 signaling at multiple levels.Cancer prevention research (Philadelphia, Pa.) · 2011Article
- c-Jun promotes whereas JunB inhibits epidermal neoplasia.The Journal of investigative dermatology · 2011Article
- Long-term type VII collagen restoration to human epidermolysis bullosa skin tissue.Human gene therapy · 2010Article
- Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice.Proceedings of the National Academy of Sciences of the United States of America · 2010Article
- The c-Jun NH2-terminal kinase 2 plays a dominant role in human epidermal neoplasia.Cancer research · 2010Article
- Article
- Defining the role of laminin-332 in carcinoma.Matrix biology : journal of the International Society for Matrix Biology · 2009Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 2 institutions in 1 country.
Funding
Abstract
The blistering disorder, lethal junctional epidermolysis bullosa (JEB), can result from mutations in the LAMB3 gene, which encodes laminin 5 beta3 (beta3). Appropriate expression of LAMbeta3 in JEB skin tissue could potentially ameliorate the symptoms of the underlying disease. To explore the utility of this therapeutic approach, primary keratinocytes from six unrelated JEB patients were transduced with a retroviral vector encoding beta3 and used to regenerate human skin on severe combined immunodeficient (SCID) mice. Tissue regenerated from beta3-transduced JEB keratinocytes produced phenotypically normal skin characterized by sustained beta3 expression and the formation of hemidesmosomes. Additionally, beta3 gene transfer corrected the distribution of a number of important basement membrane zone proteins including BPAG2, integrins beta4/beta1, and laminins alpha3/gamma2. Skin produced from beta3-negative (beta3[-]) JEB cells mimicked the hallmarks of the disease state and did not exhibit any of the aforementioned traits. Therefore, by effecting therapeutic gene transfer to beta3-deficient primary keratinocytes, it is possible to produce healthy, normal skin tissue in vivo. These data support the utility of gene therapy for JEB and highlight the potential for gene delivery in the treatment of human genetic skin disease.
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Registered trials
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.