Clinical Perspectives of Gene-Targeted Therapies for Epidermolysis Bullosa.

ABSTRACT: New insights into molecular genetics and pathomechanisms in epidermolysis bullosa (EB), methodological and technological advances in molecular biology as well as designated funding initiatives and facilitated approval procedures for orphan drugs have boosted translational research perspectives for this devastating disease. This is echoed by the increasing number of clinical trials assessing innovative molecular therapies in the field of EB. Despite remarkable progress, gene-corrective modalities, aimed at sustained or permanent restoration of functional protein expression, still await broad clinical availability. This also reflects the methodological and technological shortcomings of current strategies, including the translatability of certain methodologies beyond preclinical models as wel

SUBMITTER: Welponer T 

PROVIDER: S-EPMC8322229 | biostudies-literature | 2021 Aug

REPOSITORIES: biostudies-literature