Articles
Innovations in topical epidermolysis bullosa treatment: integrating advanced dressings, bioactive therapies and tissue-engineered skin
Daru. 2026 Apr 3;34(1):22. doi: 10.1007/s40199-026-00601-5. PMID: 41931158; PMCID: PMC13049120. Innovations in topical epidermolysis bullosa treatment: integrating advanced dressings, bioactive therapies and tissue-engineered skin Authors: Marwah […]An evaluation of prademagene zamikeracel for the treatment of recessive dystrophic epidermolysis bullosa
Expert Opin Biol Ther. 2026 Mar 18:1-6. doi: 10.1080/14712598.2026.2639393. Epub ahead of print. PMID: 41764660. An evaluation of prademagene zamikeracel for the treatment of recessive dystrophic […]Developing CRISPR-Based Therapies for Epidermolysis Bullosa: A Comprehensive Review of Current Strategies
Drugs. 2026 Feb 23. doi: 10.1007/s40265-026-02285-2. Epub ahead of print. PMID: 41731282. Developing CRISPR-Based Therapies for Epidermolysis Bullosa: A Comprehensive Review of Current Strategies Authors: du […]Efficacy and Safety of the Topical Gene Therapy Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Study of Japanese Subjects With Dystrophic Epidermolysis Bullosa
J Dermatol. 2025 Jul 16. doi: 10.1111/1346-8138.17863. Epub ahead of print. PMID: 40667654. Efficacy and Safety of the Topical Gene Therapy Beremagene Geperpavec-svdt (B-VEC) in an […]Prademagene zamikeracel for recessive dystrophic epidermolysis bullosa wounds (VIITAL): a two-centre, randomised, open-label, intrapatient-controlled phase 3 trial
Lancet. 2025 Jun 23:S0140-6736(25)00778-0. doi: 10.1016/S0140-6736(25)00778-0. Epub ahead of print. PMID: 40570869. Prademagene zamikeracel for recessive dystrophic epidermolysis bullosa wounds (VIITAL): a two-centre, randomised, open-label, intrapatient-controlled […]Targeting challenging RDEB skin wounds with gene therapy
Lancet. 2025 Jun 23:S0140-6736(25)01154-7. doi: 10.1016/S0140-6736(25)01154-7. Epub ahead of print. PMID: 40570868. Targeting challenging RDEB skin wounds with gene therapy Authors: Christen L Ebens 1 Affiliation: Abstract: […]Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa
Am J Clin Dermatol. 2025 Apr 12. doi: 10.1007/s40257-025-00942-y. Epub ahead of print. PMID: 40220208. Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label […]Splice modulation strategy applied to deep intronic variants in COL7A1 causing recessive dystrophic epidermolysis bullosa
Proc Natl Acad Sci U S A. 2024 Aug 27;121(35):e2401781121. doi: 10.1073/pnas.2401781121. Epub 2024 Aug 19. PMID: 39159368. Splice modulation strategy applied to deep intronic variants […]Emerging DNA & RNA editing strategies for the treatment of epidermolysis bullosa
J Dermatolog Treat. 2024 Dec;35(1):2391452. doi: 10.1080/09546634.2024.2391452. Epub 2024 Aug 18. PMID: 39155053. Emerging DNA & RNA editing strategies for the treatment of epidermolysis bullosa Authors: […]Highlights of Gene and Cell Therapy for Epidermolysis Bullosa and Ichthyosis
Dermatol Ther (Heidelb). 2024 Sep;14(9):2379-2392. doi: 10.1007/s13555-024-01239-4. Epub 2024 Aug 7. PMID: 39112824; PMCID: PMC11393223. Highlights of Gene and Cell Therapy for Epidermolysis Bullosa and Ichthyosis […]Treatment of Epidermolysis Bullosa and Future Directions: A Review
Dermatol Ther (Heidelb) (2024). https://doi.org/10.1007/s13555-024-01227-8 Treatment of Epidermolysis Bullosa and Future Directions: A Review Authors: Danescu S 1, Negrutiu M1, Has C2 Affiliation: Abstract: Epidermolysis bullosa (EB) […]Towards ethical drug pricing: the European Orphan Genomic Therapies Fund
Journal: Gene Ther. 2024 Apr 24. doi: 10.1038/s41434-024-00452-2. Epub ahead of print. PMID: 38658672. Towards ethical drug pricing: the European Orphan Genomic Therapies Fund Authors: Johanna […]Challenges and progress related to gene editing in rare skin diseases
Adv Drug Deliv Rev. 2024 Mar 23:115294. doi: 10.1016/j.addr.2024.115294. Epub ahead of print. PMID: 38527624. Challenges and progress related to gene editing in rare skin diseases […]Emerging Gene Therapeutics for Epidermolysis Bullosa under Development
Int J Mol Sci. 2024 Feb 13;25(4):2243. doi: 10.3390/ijms25042243. PMID: 38396920; PMCID: PMC10889532. Emerging Gene Therapeutics for Epidermolysis Bullosa under Development Authors: Bischof J, Hierl M, […]Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa
N Engl J Med. 2024 Feb 8;390(6):530-535. doi: 10.1056/NEJMoa2301244. PMID: 38324486. Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa Authors: Arianna Tovar Vetencourt 1, Ibrahim Sayed-Ahmed 1, Jennifer […]Estimated Spending on Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa
JAMA Dermatol. 2024 Jan 31. doi: 10.1001/jamadermatol.2023.5857. Epub ahead of print. PMID: 38294784; PMCID: PMC10831624. Estimated Spending on Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa Authors: Adam […]CRISPR technology: A decade of genome editing is only the beginning
Science. 2023 Jan 20;379(6629):eadd8643. doi: 10.1126/science.add8643. Epub 2023 Jan 20. PMID: 36656942. CRISPR technology: A decade of genome editing is only the beginning Authors: Joy Y […]Epidermolysis Bullosa
StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan Epidermolysis Bullosa [Updated 2024 Jan 11] Authors: Divya Khanna1; Ajoy Bardhan2 Affiliation: Abstract: No abstract available. Summary: Download: […]CRISPR/Cas9 Based Non-viral Gene Editing Therapy for Topical Treatment of Recessive Dystrophic Epidermolysis Bullosa
Molecular Therapy: Methods & Clinical Development – JOURNAL PRE-PROOF CRISPR/Cas9 Based Non-viral Gene Editing Therapy for Topical Treatment of Recessive Dystrophic Epidermolysis Bullosa Authors: Xianqing Wang 1, Xi […]Realising the potential of gene therapies for rare and ultra-rare inherited diseases
Hum Gene Ther. 2023 Aug 25. doi: 10.1089/hum.2023.127. Epub ahead of print. PMID: 37624746. Realising the potential of gene therapies for rare and ultra-rare inherited diseases […]Epidermolysis bullosa: time to come together for better outcomes
Br J Dermatol. 2023 Jul 7;189(1):5. doi: 10.1093/bjd/ljad123. PMID: 37067920. Epidermolysis bullosa: time to come together for better outcomes Authors: Gabriela Petrof 1, Anna E Martinez 1 Affiliation: Abstract: […]Heterogeneity of reported outcomes in epidermolysis bullosa clinical research: a scoping review as a first step towards outcome harmonization
Br J Dermatol. 2023 Jul 7;189(1):80-90. doi: 10.1093/bjd/ljad077. PMID: 37098154. Heterogeneity of reported outcomes in epidermolysis bullosa clinical research: a scoping review as a first step […]ABSTRACT: Compassionate use of topical beremagene geperpavec (B-VEC) for the treatment of recurrent cicatrizing conjunctivitis in a patient with dystrophic epidermolysis bullosa
Keywords: B-VEC, Krystal, Opthalmology, Gene therapyGene therapy advances shine the spotlight on epidermolysis bullosa, bringing hope to patients.
Keywords: recent different therapeutic gene therapy strategiesGene editing with ‘pencil’ rather than ‘scissors’ in human pluripotent stem cells
Keywords: Human pluripotent stem cells, Disease modeling, Ex vivo therapy, Isogenic pair, Base editors, Prime editor, Base substitution, Cas9Generation and characterization of induced pluripotent stem cell lines from two patients with recessive dystrophic epidermolysis Bullosa
Stem Cell Res. 2023 Jun;69:103104. doi: 10.1016/j.scr.2023.103104. Epub 2023 Apr 24. PMID: 37148821. Generation and characterization of induced pluripotent stem cell lines from two patients with […]A Review of CRISPR-Based Advances in Dermatological Diseases
Mol Diagn Ther. 2023 Jul;27(4):445-456. doi: 10.1007/s40291-023-00642-5. Epub 2023 Apr 11. PMID: 37041404. A Review of CRISPR-Based Advances in Dermatological Diseases Authors: Lu X, Jin H. […]Stairways to Advanced Therapies for Epidermolysis Bullosa
Cold Spring Harb Perspect Biol. 2023 Apr 3;15(4):a041229. doi: 10.1101/cshperspect.a041229. PMID: 36167646; PMCID: PMC10071437 Stairways to Advanced Therapies for Epidermolysis Bullosa Authors: Laura De Rosa # 1, Elena Enzo # 2, Michele […]The steep uphill path leading to ex vivo gene therapy for genodermatoses
Am J Physiol Cell Physiol. 2022 Sep 1;323(3):C896-C906. doi: 10.1152/ajpcell.00117.2022. Epub 2022 Aug 1. PMID: 35912986. The steep uphill path leading to ex vivo gene therapy […]The joint battle to tackle epidermolysis bullosa through gene therapy
Trends Mol Med. 2022 Jul;28(7):533-535. doi: 10.1016/j.molmed.2022.05.001. Epub 2022 May 19. PMID: 35599142. The joint battle to tackle epidermolysis bullosa through gene therapy Authors: Laura De […]Clonal analysis of human clonogenic keratinocytes
Methods Cell Biol. 2022;170:101-116. doi: 10.1016/bs.mcb.2022.02.009. Epub 2022 Apr 18. PMID: 35811094. Clonal analysis of human clonogenic keratinocytes Authors: Elena Enzo 1, Camilla Cattaneo 1, Federica Consiglio 2, Maria Pia Polito 1, Sergio Bondanza 2, Michele […]In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial
Nat Med. 2022 Apr;28(4):780-788. doi: 10.1038/s41591-022-01737-y. Epub 2022 Mar 28. PMID: 35347281; PMCID: PMC9018416. In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase […]A non-viral and selection-free COL7A1 HDR approach with improved safety profile for dystrophic epidermolysis bullosa
Mol Ther Nucleic Acids. 2021 May 29;25:237-250. doi: 10.1016/j.omtn.2021.05.015. PMID: 34458008; PMCID: PMC8368800. A non-viral and selection-free COL7A1 HDR approach with improved safety profile for dystrophic epidermolysis bullosa […]EBGene trial: patient preselection outcomes for the European GENEGRAFT ex vivo phase I/II gene therapy trial for recessive dystrophic epidermolysis bullosa
Br J Dermatol. 2020 Mar;182(3):794-797. doi: 10.1111/bjd.18559. Epub 2019 Nov 27. PMID: 31557321. EBGene trial: patient preselection outcomes for the European GENEGRAFT ex vivo phase I/II gene […]