Gene Therapy
1 March 2020

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 […]
29 May 2021

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 […]
28 March 2022

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 […]
11 April 2023

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. […]
1 June 2023

Generation 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 […]
20 June 2023

Gene 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, Cas9
26 June 2023

Gene therapy advances shine the spotlight on epidermolysis bullosa, bringing hope to patients.

Keywords: recent different therapeutic gene therapy strategies
4 July 2023

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 therapy
7 July 2023

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 […]