The DOI matches its Crossref record; 39 of the 39 references it cites were found in a registry or an index.
Human COL7A1 -corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa
Open accessAbstract
Patient-specific iPSCs were derived, corrected, and differentiated into human skin grafts for the treatment of recessive dystrophic epidermolysis bullosa.
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“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]
Experimental — accuracy not yet measuredIn the citing paper
“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]
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“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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Research works printed but not linked: 1
- Siprashvili Z., Nguyen N., Gorell E., Khuu P., Furukawa L., Lorenz H., Leung T., Keene D., Khavari P., Marinkovich M., Lane A., Phase I clinical trial of genetically corrected autologous epidermal keratinocytes for recessive dystrophic epidermolysis bullosa. J. Invest. Dermatol. 134, S75 (2014).
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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).”
Background
Metadata
- 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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