[Genome Editing Tools and their Application in Experimental Ophthalmology].

Area of Science:

• Molecular Biology
• Genetics
• Ophthalmology

Background:

• Genome editing tools, including zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN), and CRISPR/Cas systems, offer precise DNA cleavage.
• DNA double-strand breaks are repaired via non-homologous end joining (NHEJ) and homology-directed repair (HDR).

Purpose of the Study:

• To explore the revolutionary impact of genome editing technologies on molecular biology and experimental ophthalmology.
• To highlight the potential of gene editing for therapeutic strategies targeting retinal diseases.

Main Methods:

• Utilizing CRISPR/Cas systems for site-specific DNA cleavage in living cells.
• Employing NHEJ and HDR repair pathways with DNA templates to correct gene defects or mutations.

Main Results:

• CRISPR/Cas systems demonstrate high efficiency in site-specific DNA cleavage.
• Genome editing facilitates gene knockout and mutation repair through cellular repair pathways.

Conclusions:

• Genome editing technologies provide new strategies for basic, biotechnological, and biomedical sciences.
• The eye's accessibility makes retinal diseases a primary focus for in vivo therapeutic gene editing.