RPGR gene therapy presents challenges in cloning the coding sequence.

ABSTRACT: Introduction: Currently, there are three Phase I/II clinical trials based on gene therapy ongoing to test different AAV.RPGR or deleted RPGR vectors on patients affected by X-linked retinitis pigmentosa. These three vectors differ in the adeno-associated viral (AAV) vector capsid used, and the coding sequences: two contain codon optimized versions of RPGR which give the full-length protein, whilst the third uses a wild-type sequence that contains a large deletion encoding part of the functional domain of the RPGR protein.Areas covered: This review approaches the different studies that have led to the initiation of three different clinical trials for RPGR related X-linked retinitis pigmentosa.Expert opinion: The development of a gene therapy vector to deli

SUBMITTER: Martinez-Fernandez De La Camara C 

PROVIDER: S-EPMC7104355 | biostudies-literature | 2020 Jan

REPOSITORIES: biostudies-literature