Advanced Research on Retina: Recent Development of Genome Editing Strategies Aimed at Treating Inherited Retinal Diseases

Dear Colleagues,

This Special Issue of Cells focuses on inherited retinal diseases, a genetically diverse group of blinding disorders primarily characterized by photoreceptor neurodegeneration. Affecting approximately 1 in 3,000 individuals globally, these conditions significantly impair patients' quality of life. Over 270 causative genes have been identified, driving intense interest in gene therapy utilizing adeno-associated viruses (AAVs) to deliver wildtype copies and counteract loss-of-function mutations. However, this approach faces challenges such as unmet physiological demand from exogenously expressed complementing genes, transgene silencing within AAV vectors, and persistent stress from unrepaired mutant alleles.

Recent advancements in genome editing offer promising avenues to address the root causes of genetic disorders. Clinical trials employing CRISPR-Cas-based technologies are already underway, including for inherited retinal diseases. This Special Issue aims to present an encompassing view of genome editing efforts targeting inherited retinal diseases. It emphasizes the creation of animal models that validate proof-of-concept strategies and explores DNA damage repair pathways implicated in genome editing across distinct eye tissue compartments. By highlighting these aspects, the issue contributes to advancing our understanding and therapeutic approaches for inherited retinal diseases.

Dr. Lijin Dong
Guest Editor

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• retina
• RPE
• neurodegeneration
• retina neurodegeneration
• retinitis pigmentosa
• genome editing
• CRISPR
• DNA double strand break
• DNA repair
• homologous recombination
• indel