Publications
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 […]
1 April 2021

Integrin α3β1 Is a Key Regulator of Several Protumorigenic Pathways during Skin Carcinogenesis

 J Invest Dermatol. 2021 Apr;141(4):732-741.e6. doi: 10.1016/j.jid.2020.07.024. Epub 2020 Aug 14. PMID: 32805217. Integrin α3β1 Is a Key Regulator of Several Protumorigenic Pathways during Skin Carcinogenesis […]
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 […]
5 September 2022

Stanford Adult Epidermolysis Bullosa Anesthesia Protocol 

PDF Stanford Adult Epidermolysis Bullosa Anesthesia Protocol  Authors:  Erin Bushell, MD and Brita Mittal, MD   Department of Anesthesiology, Perioperative, and Pain Medicine, Stanford University School of […]
29 March 2023

Mapping the burden of severe forms of epidermolysis bullosa – Implications for patient management

JAAD Int. 2023 Mar 29;11:224-232. doi: 10.1016/j.jdin.2023.02.016. PMID: 37179539; PMCID: PMC10173168. Mapping the burden of severe forms of epidermolysis bullosa – Implications for patient management Authors: […]
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. […]
17 April 2023

How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis

Orphanet J Rare Dis. 2023 Apr 17;18(1):84. doi: 10.1186/s13023-023-02682-w. PMID: 37069597; PMCID: PMC10108537. How do patients and other members of the public engage with the orphan […]