Clustered Regularly Interspaced Short Palindromic Repeats-Based Genome Surgery for the Treatment of Autosomal Dominant Retinitis Pigmentosa.

ABSTRACT:

Purpose

To develop a universal gene therapy to overcome the genetic heterogeneity in retinitis pigmentosa (RP) resulting from mutations in rhodopsin (RHO).

Design

Experimental study for a combination gene therapy that uses both gene ablation and gene replacement.

Participants

This study included 2 kinds of human RHO mutation knock-in mouse models: Rho^P23H and Rho^D190N. In total, 23 Rho^P23H/P23H, 43 Rho^P23H/+, and 31 Rho^D190N/+ mice were used for analysis.

Methods

This study involved gene therapy using dual adeno-associated viruses (AAVs) that (1) destroy expression of the endogenous Rho gene in a mutation-independent manner via an improved clustered regularly interspaced short palindromic repeats-based gene

SUBMITTER: Tsai YT 

PROVIDER: S-EPMC6109419 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature