ArticleScience translational medicine2014
Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa.
Article in Science translational medicine, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 102 papers, 2 of them syntheses that pooled it.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
102 citing papers in PubMed, 2 syntheses or guidelines pooled it, 213 citations in OpenAlex.
- The Efficacy of Stem Cells in Wound Healing: A Systematic Review.International journal of molecular sciences · 2024Pooled it
- A Systematic Review of Stem Cell Differentiation into Keratinocytes for Regenerative Applications.Stem cell reviews and reports · 2024Pooled it
- Phase 1/2a clinical trial of gene-corrected autologous cell therapy for recessive dystrophic epidermolysis bullosa.JCI insight · 2019Trial
- YAP-TEAD regulates the super-enhancer network to control early surface ectoderm commitment.Nucleic acids research · 2026Article
- Engineering the next generation of allogeneic CAR cells: iPSCs as a scalable and editable platform.Stem cell reports · 2025Review
- Recent progress in the identification and in vitro culture of skin organoids.Regenerative therapy · 2025Review
- Genome editing strategies for targeted correction of β-globin mutation in sickle cell disease: From bench to bedside.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- A spatiotemporal and machine-learning platform facilitates the manufacturing of hPSC-derived esophageal mucosa.Developmental cell · 2025Article
- Cell Therapy and the Skin: Great Potential but in Need of Optimization.The Journal of investigative dermatology · 2025Review
- Considerations for the development of iPSC-derived cell therapies: a review of key challenges by the JSRM-ISCT iPSC Committee.Cytotherapy · 2024Review
- A scalable and cGMP-compatible autologous organotypic cell therapy for Dystrophic Epidermolysis Bullosa.Nature communications · 2024Article
- Hippo cooperates with p53 to maintain foregut homeostasis and suppress the malignant transformation of foregut basal progenitor cells.Proceedings of the National Academy of Sciences of the United States of America · 2024Article
- Advances in Genetic Reprogramming: Prospects from Developmental Biology to Regenerative Medicine.Current medicinal chemistry · 2024Review
- A Review of CRISPR-Based Advances in Dermatological Diseases.Molecular diagnosis & therapy · 2023Review
- Engineering edgeless human skin with enhanced biomechanical properties.Science advances · 2023Article
- iPSC-based approach for human hair follicle regeneration.Frontiers in cell and developmental biology · 2023Review
- Extracellular vesicles modulate key signalling pathways in refractory wound healing.Burns & trauma · 2023Review
- Directed differentiation of human pluripotent stem cells into epidermal keratinocyte-like cells.STAR protocols · 2022Article
- Genetic Disorders of the Extracellular Matrix: From Cell and Gene Therapy to Future Applications in Regenerative Medicine.Annual review of genomics and human genetics · 2022Review
- Article
42 more citing papers are in PubMed but not listed here.
Corrections and comments
- Erratum issuedDerafshi, Bahareh Haddad [corrected to Haddad, Bahareh]
Authors and funding
23 authors at 1 institution in 1 country.
Funding
Abstract
Patients with recessive dystrophic epidermolysis bullosa (RDEB) lack functional type VII collagen owing to mutations in the gene COL7A1 and suffer severe blistering and chronic wounds that ultimately lead to infection and development of lethal squamous cell carcinoma. The discovery of induced pluripotent stem cells (iPSCs) and the ability to edit the genome bring the possibility to provide definitive genetic therapy through corrected autologous tissues. We generated patient-derived COL7A1-corrected epithelial keratinocyte sheets for autologous grafting. We demonstrate the utility of sequential reprogramming and adenovirus-associated viral genome editing to generate corrected iPSC banks. iPSC-derived keratinocytes were produced with minimal heterogeneity, and these cells secreted wild-type type VII collagen, resulting in stratified epidermis in vitro in organotypic cultures and in vivo in mice. Sequencing of corrected cell lines before tissue formation revealed heterogeneity of cancer-predisposing mutations, allowing us to select COL7A1-corrected banks with minimal mutational burden for downstream epidermis production. Our results provide a clinical platform to use iPSCs in the treatment of debilitating genodermatoses, such as RDEB.
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What OpenQuestion holds
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.