Articles
CRISPR/Cas9 Based Non-viral Gene Editing Therapy for Topical Treatment ofRecessive Dystrophic Epidermolysis Bullosa
Molecular Therapy: Methods & Clinical Development – JOURNAL PRE-PROOF CRISPR/Cas9 Based Non-viral Gene Editing Therapy for Topical Treatment ofRecessive Dystrophic Epidermolysis Bullosa Authors: Xianqing Wang, 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. […]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 […]