On August 9th, 2021, recombinant adeno-associated virus vector carrying human CYP4V2 gene has obtained Orphan Drug Designations and Approvals by the US FDA. This gene therapy is offered by China Gene technology. It brings hope for RP patients with the gene mutation of CYP4V2, which is also called Bietti crystalline dystrophy.
Retinitis pigmentosa, congenital macular dystrophy, optic nerve atrophy and other congenital hereditary eye disorders are like a train rushing to the darkness. If not treated, they will eventually lead to blindness.
Gene therapy is like the brakes of this train. It can stop the train, prevent him from continuing to run into the darkness, slow down the vision loss process, solve the problem of genetic mutation, and fundamentally solve the cause of the problem.
Stem cell therapy is to pull the train back to escape the darkness and improve eyesight. However, stem cells are not done once and for all. Once the effect disappears, the train will run into the darkness again.
Therefore, the treatment of retinitis pigmentosa and other optic neuropathy, ultimately requires gene therapy to stabilize the disease, and stem cell therapy to improve vision. In daily life, some anti-inflammatory, anti-oxidative and anti-apoptotic supplements will also benefit the maintenance of the visual function. We believe it will not take long until we find the final solution for RP patients to regain their vision!