ReviewInternational journal of molecular sciences2024
Emerging Gene Therapeutics for Epidermolysis Bullosa under Development.
Review in International journal of molecular sciences, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
17 citing papers in PubMed, 26 citations in OpenAlex.
- From the Microscope to the Genome: A New Era in the Molecular Genetics of Epidermolysis Bullosa.International journal of molecular sciences · 2026Review
- Epidermolysis bullosa and global challenges in translational research for rare diseases.Einstein (Sao Paulo, Brazil) · 2026Article
- Developing CRISPR-Based Therapies for Epidermolysis Bullosa: A Comprehensive Review of Current Strategies.Drugs · 2026Review
- Gene therapy for genodermatoses at the crossroads of innovation and clinical translation.Frontiers in bioengineering and biotechnology · 2026Review
- Landscape ofMolecular therapy. Methods & clinical development · 2025Article
- Off-label use of recombinant adjuvanted Herpes Zoster vaccine in a 10-year-old high-risk patient affected by epidermolysis bullosa: A case report.Human vaccines & immunotherapeutics · 2025Article
- Efficient Dual Cas9 Nickase Correction of a Prevalent PathogenicJID innovations : skin science from molecules to population health · 2025Article
- Enhanced HDR-mediated correction of heterozygousMolecular therapy. Nucleic acids · 2025Article
- Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives.Pharmaceutics · 2025Review
- Epidermolysis Bullosa with Esophageal Complications and Co-Infection withInfection and drug resistance · 2025Article
- Advances in regenerative medicine-based approaches for skin regeneration and rejuvenation.Frontiers in bioengineering and biotechnology · 2025Review
- Gene hydrogel platforms for targeted skin therapy: bridging hereditary disorders, chronic wounds, and immune related skin diseases.Frontiers in drug delivery · 2025Review
- Advancing rare disease therapeutics through digital twins: Opportunities in drug development and precision dosing.Computational and structural biotechnology journal · 2025Review
- Advances in Microengineered Platforms for Skin Research.JID innovations : skin science from molecules to population health · 2025Review
- Treatment of Epidermolysis Bullosa and Future Directions: A Review.Dermatology and therapy · 2024Review
- Genetic Implications and Management of Epidermolysis Bullosa in the Saudi Arabian Population.Cureus · 2024Review
- Epidermolysis Bullosa: Two rare case reports of COL7A1 and EBS-GEN SEV KRT14 variants with review of literature.BMC pediatrics · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
Abstract
The monogenetic disease epidermolysis bullosa (EB) is characterised by the formation of extended blisters and lesions on the patient's skin upon minimal mechanical stress. Causal for this severe condition are genetic mutations in genes, leading to the functional impairment, reduction, or absence of the encoded protein within the skin's basement membrane zone connecting the epidermis to the underlying dermis. The major burden of affected families justifies the development of long-lasting and curative therapies operating at the genomic level. The landscape of causal therapies for EB is steadily expanding due to recent breakthroughs in the gene therapy field, providing promising outcomes for patients suffering from this severe disease. Currently, two gene therapeutic approaches show promise for EB. The clinically more advanced gene replacement strategy was successfully applied in severe EB forms, leading to a ground-breaking in vivo gene therapy product named beremagene geperpavec (B-VEC) recently approved from the US Food and Drug Administration (FDA). In addition, the continuous innovations in both designer nucleases and gene editing technologies enable the efficient and potentially safe repair of mutations in EB in a potentially permanent manner, inspiring researchers in the field to define and reach new milestones in the therapy of EB.
Indexed as
Identifiers
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.