Mutation-Independent Therapies for Retinal Diseases: Focus on Gene-Based Approaches.

ABSTRACT: Gene therapy is proving to be an effective approach to treat or prevent ocular diseases ensuring a targeted, stable, and regulated introduction of exogenous genetic material with therapeutic action. Retinal diseases can be broadly categorized into two groups, namely monogenic and complex (multifactorial) forms. The high genetic heterogeneity of monogenic forms represents a significant limitation to the application of gene-specific therapeutic strategies for a significant fraction of patients. Therefore, mutation-independent therapeutic strategies, acting on common pathways that underly retinal damage, are gaining interest as complementary/alternative approaches for retinal diseases. This review will provide an overview of mutation-independent strategies that rely on the modulation in the r

SUBMITTER: Carrella S 

PROVIDER: S-EPMC7541846 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature