Gene therapy for the treatment of X-linked retinitis pigmentosa.

ABSTRACT:

Introduction

X-linked retinitis pigmentosa caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene is the most common form of recessive RP. The phenotype is characterised by its severity and rapid disease progression. Gene therapy using adeno-associated viral vectors is currently the most promising therapeutic approach. However, the construction of a stable vector encoding the full-length RPGR transcript has previously proven to be a limiting step towards gene therapy clinical trials. Recently however, a codon optimised version of RPGR has been shown to increase the stability and fidelity of the sequence, conferring a therapeutic effect in murine and canine animal models.

Areas covered

This manuscript reviews the natural history of X

SUBMITTER: Martinez-Fernandez De La Camara C 

PROVIDER: S-EPMC6059358 | biostudies-literature | 2018 Feb

REPOSITORIES: biostudies-literature