Clinical Perspectives of Gene-Targeted Therapies for Epidermolysis Bullosa.
Dermatol Ther (Heidelb)
; 11(4): 1175-1197, 2021 Aug.
Article
em En
| MEDLINE
| ID: mdl-34110606
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 well as the safe, specific, efficient, feasible, sustained and cost-effective delivery of therapeutic/corrective information to target cells. This review gives an updated overview on status, prospects, challenges and limitations of current gene-targeted therapies.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Idioma:
En
Revista:
Dermatol Ther (Heidelb)
Ano de publicação:
2021
Tipo de documento:
Article
País de afiliação:
Áustria