Ocular gene therapy targeting retinal angiogenesis and vascular leakage: translational and clinical evidence from a systematic review and meta-analysis.

In 30 seconds

This systematic review and meta-analysis evaluated ocular gene therapy's effects on retinal angiogenesis and vascular leakage in various studies. It found that preclinical gene therapy significantly reduced pathological neovascularization (SMD -1.16) and vascular leakage (MD 0.215). In clinical cohorts, gene therapy reduced central subfield thickness by -55.30 µm and decreased anti-VEGF treatment burden, with approximately 65% of participants needing less supplemental anti-VEGF.

Key findings

• Preclinical gene therapy reduced pathological neovascularization with a pooled SMD of -1.16.
• Vascular leakage was significantly reduced with a pooled MD of 0.215.
• Clinical gene therapy reduced central subfield thickness by -55.30 µm.
• Approximately 65% of participants experienced reduced anti-VEGF treatment burden.

Why it matters

The findings suggest that ocular gene therapy could offer a more durable treatment option for retinal diseases, potentially reducing the need for frequent anti-VEGF injections and improving patient outcomes.