Topical Gene Therapy for Epidermolysis Bullosa

In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial

(2022) Irina Gurevich et al.   NATURE MEDICINE

295 Collagen 7 (C7) protein replacement therapy (PTR-01) durably reduces wound size and symptoms in patients with recessive dystrophic epidermolysis bullosa (RDEB)

(2022) A. Bruckner et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa: a preclinical study

(2020) Olivier Bornert et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Epidermolysis bullosa

(2020) Ajoy Bardhan et al.   Nature Reviews Disease Primers

591 A phase 1/2 study of genetically-corrected, collagen VII expressing autologous human dermal fibroblasts injected into the skin of patients with recessive dystrophic epidermolysis bullosa (RDEB)

(2018) M. Marinkovich et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Gentamicin induces functional type VII collagen in recessive dystrophic epidermolysis bullosa patients

(2017) David T. Woodley et al.   JOURNAL OF CLINICAL INVESTIGATION

Epidemiology of Inherited Epidermolysis Bullosa Based on Incidence and Prevalence Estimates From the National Epidermolysis Bullosa Registry

(2016) Jo-David Fine   JAMA Dermatology