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Retinitis Pigmentosa: From Pathomolecular Mechanisms to Therapeutic Strategies
Enzo Maria Vingolo1, Simona Mascolo1, Filippo Miccichè1
1Sense Organs Department, UOSD of Ophtalmology, University la Sapienza of Rome, Polo Pontino-Ospedale A. Fiorini, 4019 Terracina, Italy.
Abstract:
Retinitis pigmentosa is an inherited disease, in which mutations in different types of genes lead to the death of photoreceptors and the loss of visual function. Although retinitis pigmentosa is the most common type of inherited retinal dystrophy, a clear line of therapy has not yet been defined. In this review, we will focus on the therapeutic aspect and attempt to define the advantages and disadvantages of the protocols of different therapies. The role of some therapies, such as antioxidant agents or gene therapy, has been established for years now. Many clinical trials on different genes and mutations causing RP have been conducted, and the approval of voretigene nepavorec by the FDA has been an important step forward. Nonetheless, even if gene therapy is the most promising type of treatment for these patients, other innovative strategies, such as stem cell transplantation or hyperbaric oxygen therapy, have been shown to be safe and improve visual quality during clinical trials. The treatment of this disease remains a challenge, to which we hope to find a solution as soon as possible.
Insights
Retinitis pigmentosa (RP) treatments are evolving, with gene therapy showing promise. Innovative strategies like stem cell therapy and hyperbaric oxygen also offer potential for improving vision in inherited retinal dystrophies.
Area of Science:
- Ophthalmology
- Genetics
- Regenerative Medicine
Background:
- Retinitis pigmentosa (RP) is a common inherited retinal dystrophy causing photoreceptor death and vision loss.
- Despite advances, a definitive therapy for RP remains undefined.
- Mutations in various genes contribute to the diverse clinical presentations of RP.
Purpose of the Study:
- To review and analyze the therapeutic landscape for retinitis pigmentosa.
- To evaluate the advantages and disadvantages of different treatment protocols.
- To highlight recent advancements and future directions in RP therapy.
Main Methods:
- Review of established and emerging therapies for retinitis pigmentosa.
- Analysis of clinical trial data for gene therapy, stem cell transplantation, and hyperbaric oxygen therapy.
- Discussion of the established roles of antioxidant agents and gene therapy.
Main Results:
- Gene therapy, exemplified by voretigene nepavorec approval, represents a significant therapeutic advancement.
- Stem cell transplantation and hyperbaric oxygen therapy have demonstrated safety and visual function improvements in trials.
- Established therapies like antioxidant agents and gene therapy continue to be investigated.
Conclusions:
- Gene therapy is a highly promising treatment for retinitis pigmentosa patients.
- Innovative approaches such as stem cell transplantation and hyperbaric oxygen therapy offer additional therapeutic avenues.
- Developing effective treatments for retinitis pigmentosa remains a critical challenge in ophthalmology.
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