The Landscape of Non-Viral Gene Augmentation Strategies for Inherited Retinal Diseases.

ABSTRACT: Inherited retinal diseases (IRDs) are a heterogeneous group of disorders causing progressive loss of vision, affecting approximately one in 1000 people worldwide. Gene augmentation therapy, which typically involves using adeno-associated viral vectors for delivery of healthy gene copies to affected tissues, has shown great promise as a strategy for the treatment of IRDs. However, the use of viruses is associated with several limitations, including harmful immune responses, genome integration, and limited gene carrying capacity. Here, we review the advances in non-viral gene augmentation strategies, such as the use of plasmids with minimal bacterial backbones and scaffold/matrix attachment region (S/MAR) sequences, that have the capability to overcome these weaknesses by accommodating genes

SUBMITTER: Toualbi L 

PROVIDER: S-EPMC7956638 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature