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Mutations in the  RPGR  gene are responsible for one of the most prevalent and severe types of retinitis pigmentosa. Gene therapy has shown great promise to treat inherited retinal diseases, and currently, four RPGR gene therapy vectors are being evaluated in clinical trials. This manuscript reviews the gene therapy products that are in development for X-linked retinitis pigmentosa caused by mutations in  RPGR , and the challenges that scientists and clinicians have faced. The development of a gene therapy product for  RPGR -associated retinal degeneration has been a great challenge due to the incomplete understanding of the underlying genetics and mechanism of action of RPGR, and on the other hand, due to the instability of the  RPGR  gene. Three of the four gene therapy vectors currently in clinical trials include a codon-optimized version of the human  RPGR  sequence, and the other vector contains a shortened version of the human  RPGR . To date, the only Phase I/II results published in a peer-reviewed journal demonstrate a good safety profile and an improvement in the visual field using a codon optimized version of  RPGRORF15.

Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa