Human COL7A1 -corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa
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Patient-specific iPSCs were derived, corrected, and differentiated into human skin grafts for the treatment of recessive dystrophic epidermolysis bullosa.
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Skin organoids can be readily generated from patient-derived cells and used to model genetic disorders, such as epidermolysis bullosa.
“Furthermore, skin organoids can be readily generated from patient-derived cells and used to model genetic disorders, such as epidermolysis bullosa”
From Generation and characterization of hair-bearing skin organoids from human pluripotent stem cells (2022) · cites this paper as [60]
The Abstract and Methods describe generating stratified epidermis in organotypic cultures from RDEB patient-derived, gene-corrected cells. They do not describe skin organoids used to model the disorder, and the Results note variability between cell lines rather than establishing that generation is readily achieved. Page numbers are unavailable in our copy. (LLM, conf=0.86)
Experimental — accuracy not yet measuredHuman skin equivalents can be created entirely from fibroblasts and keratinocytes generated from patients' iPSCs.
“It has been shown that human skin equivalents can be created entirely from fibroblasts and keratinocytes generated from patients’ iPSCs, and that healthy cells can be generated from reprogrammed cells from patients with RDEB (153, 154).”
From Wound repair and regeneration: Mechanisms, signaling, and translation (2014) · cites this paper as [154]
Healthy cells can be generated from reprogrammed cells from patients with RDEB.
“and that healthy cells can be generated from reprogrammed cells from patients with RDEB (153, 154).”
From Wound repair and regeneration: Mechanisms, signaling, and translation (2014) · cites this paper as [154]
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“, 2016) and to derive isogeneic cell pairs of COL7A1-corrected iPSCs derived from patients with dystrophic epidermolysis bullosa (Sebastiano et al., 2014).”
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- Authors
- Sebastiano, V., Zhen, H. H., Haddad, B., Bashkirova, E., Melo, S. P., Wang, P., Leung, T. L., Siprashvili, Z., Tichy, A., Li, J., Ameen, M., Hawkins, J., Lee, S., Li, L., Schwertschkow, A., Bauer, G., Lisowski, L., Kay, M. A., Kim, S. K., Lane, A. T., Wernig, M., & Oro, A. E.
- Affiliations
- Institute for Stem Cell Biology and Regenerative Medicine (US)
- Stanford Medicine (US)
- Stanford University (US)
- University of California, Davis (US)
- Corresponding author
- Sebastiano, Vittorio
- Zhen, Hanson Hui
- Wernig, Marius
- Oro, Anthony E.
- ORCID
- Venue
- Science Translational Medicine
- Published
- 2014-11-26
- Volume
- 6
- Issue
- 264
- Pages
- 264ra163-264ra163
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- PMC
- PMC4428910
- OpenAlex
- W2085600205
- Cited by
- 213
- References
- 39
- Citations by year
- 2026: 2 · 2025: 10 · 2024: 9 · 2023: 12 · 2022: 17 · 2021: 29 · 2020: 15 · 2019: 21 · 2018: 23 · 2017: 27 · 2016: 22 · 2015: 23 · 2014: 3
- FWCI
- 10.26
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- Howard Hughes Medical Institute
- National Heart, Lung, and Blood Institute (R01HL064274)
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (R01AR055914, K08AR066661, T32AR007422)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Pluripotent Stem Cells Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Cell Biology › Skin and Cellular Biology Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Keywords
- recessive dystrophic epidermolysis bullosa, COL7A1, type VII collagen, induced pluripotent stem cells, genome editing, keratinocytes, organotypic culture, genodermatosis, squamous cell carcinoma
- MeSH terms
- Animals, Base Sequence, Genes, Recessive, Humans, Molecular Sequence Data, Mutation, Genetic Therapy, Keratinocytes, Genome, Human, Epidermolysis Bullosa Dystrophica, Sequence Analysis, DNA, Genetic Predisposition to Disease, Collagen Type VII, Mice, Induced Pluripotent Stem Cells, Homologous Recombination
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