Corneal gene therapy: Structural and mechanistic understanding.

Area of Science:

• Ophthalmology
• Molecular Biology
• Regenerative Medicine

Background:

• Corneal diseases are a leading cause of vision impairment globally.
• Current treatments like small-molecule drugs and corneal transplants have limitations.
• There is a critical need for novel, non-surgical therapies for corneal disorders.

Purpose of the Study:

• To review the structural and functional aspects of the cornea.
• To elucidate the mechanisms of gene therapy vectors and gene editing techniques.
• To discuss the current status of gene therapy for corneal diseases and genetic dystrophies.

Main Methods:

• Review of existing literature on corneal biology and gene therapy.
• Analysis of gene editing technologies (e.g., CRISPR-Cas9).
• Evaluation of various gene delivery vectors (viral and non-viral).

Main Results:

• Gene therapy presents a viable alternative to traditional treatments for corneal blindness.
• Careful selection of genes, editing methods, and delivery vectors is crucial for therapeutic success.
• Gene therapy holds potential for safe, sustained, and non-immunogenic treatment responses.

Conclusions:

• Gene therapy is a highly promising strategy for treating corneal disorders and genetic dystrophies.
• Advancements in gene editing and delivery systems are key to realizing its full potential.
• Further research is needed to optimize gene therapy protocols for clinical application in ophthalmology.