Leading edge: emerging drug, cell, and gene therapies for junctional epidermolysis bullosa.

ABSTRACT:

Introduction

Junctional epidermolysis bullosa (JEB) is a rare inherited genetic disorder with limited treatments beyond palliative care. A major hallmark of JEB is skin blistering caused by functional loss or complete absence of major structural proteins of the skin. Impaired wound healing in patients with JEB gives rise to chronic cutaneous ulcers that require daily care. Wound care and infection control are the current standard of care for this patient population.

Areas covered

This review covers research and clinical implementation of emerging drug, cell, and gene therapies for JEB. Current clinical trials use topical drug delivery to manipulate the inflammation and re-epithelialization phases of wound healing or promote premature stop codon readthrough to accelerate chro

SUBMITTER: Keith AR 

PROVIDER: S-EPMC7392816 | biostudies-literature | 2020 Aug

REPOSITORIES: biostudies-literature