Retinitis Pigmentosa and Therapeutic Approaches: A Systematic Review

Area of Science:

• Ophthalmology
• Genetics
• Regenerative Medicine

Background:

• Retinitis pigmentosa (RP) encompasses hereditary retinal dystrophies causing progressive photoreceptor degeneration and severe vision loss.
• Current management for RP lacks definitive cures, necessitating exploration of novel therapeutic strategies.

Purpose of the Study:

• To systematically review the efficacy and safety of emerging treatments for retinitis pigmentosa (RP).
• To evaluate gene therapy, mesenchymal cell-based approaches, and supplementary interventions for RP.

Main Methods:

• Comprehensive literature search of electronic databases up to February 2024.
• Inclusion of randomized controlled trials, non-randomized studies, and case series reporting RP treatment outcomes.
• Data extraction and synthesis focused on evidence quality and key findings.

Main Results:

• Thirteen studies met inclusion criteria, evaluating diverse RP treatment modalities.
• Gene therapy demonstrated promise in preserving visual function and stabilizing RP progression.
• Mesenchymal cell therapies showed potential but with limited and heterogeneous evidence; supplementary interventions had variable efficacy.

Conclusions:

• Emerging RP treatments offer potential for slowing disease progression and preserving vision, though not curative.
• Significant evidence gaps and methodological heterogeneity necessitate further research for optimal RP treatment strategies.
• Future research should focus on refining patient selection and enhancing long-term outcomes for RP management.