Precision Is Not Enough: When Tools Outpace Translation in Ocular Gene Therapy

Area of Science:

• Ophthalmology
• Molecular Biology
• Genetics

Background:

• The eye's unique characteristics, including surgical accessibility and immune privilege, make it an ideal site for gene therapy.
• The contralateral eye serves as a valuable anatomical control in ocular gene therapy studies.

Purpose of the Study:

• To review the historical development of gene discovery relevant to ocular conditions.
• To synthesize current gene therapy strategies for inherited and acquired eye diseases.
• To critically assess the limitations of CRISPR and genome-editing technologies in ophthalmology.

Main Methods:

• Historical review of gene discovery.
• Synthesis of current gene therapy approaches for ocular disorders.
• Critical evaluation of genome-editing technologies like CRISPR.
• Examination of scientific and translational challenges.

Main Results:

• The eye is a prime location for gene therapy due to its accessibility and immune privilege.
• Current gene therapy strategies show promise for various ocular conditions.
• CRISPR and related technologies have limitations that need addressing for clinical application.

Conclusions:

• Integrating genetic therapies into routine ophthalmic practice requires overcoming key scientific and translational hurdles.
• Further research is needed to refine genome-editing tools and address delivery and safety concerns.
• The eye remains a leading platform for advancing gene therapy applications.