Developing Non-Human Primate Models of Inherited Retinal Diseases

Area of Science:

• Ophthalmology
• Genetics
• Translational Medicine

Background:

• Inherited retinal diseases (IRDs) are a diverse group of genetic disorders leading to blindness.
• Advances in sequencing have improved understanding and genotype-phenotype correlations in IRDs.
• Therapeutic development for IRDs is rapidly advancing.

Purpose of the Study:

• To review available non-human primate (NHP) models for inherited retinal diseases.
• To discuss methods for developing NHP IRD models, emphasizing gene-editing technologies.
• To address the challenges in creating NHP models that accurately mimic human IRD phenotypes.

Main Methods:

• Review of existing literature on NHP models for IRDs.
• Analysis of gene-editing technologies (e.g., CRISPR-Cas9) used in NHP model development.
• Discussion of anatomical and genetic similarities between NHPs and humans relevant to IRDs.

Main Results:

• Several NHP IRD models are currently available.
• Gene-editing technologies offer promising avenues for creating specific IRD phenotypes in NHPs.
• Technical and financial challenges remain in developing and utilizing NHP models.

Conclusions:

• NHP models are valuable for preclinical assessment of IRD therapies due to their human-like similarities.
• Gene-editing advancements are crucial for refining NHP models to better recapitulate specific IRDs.
• Further research and development are needed to overcome challenges in NHP IRD model creation and application.