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 […]
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 […]
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
25 August 2023

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 […]
11 October 2023

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 […]
31 January 2024

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 […]