Developing Non-Human Primate Models of Inherited Retinal Diseases

Genes (Basel). 2022 Feb 14;13(2):344. doi: 10.3390/genes13020344.

Abstract

Inherited retinal diseases (IRDs) represent a genetically and clinically heterogenous group of diseases that can eventually lead to blindness. Advances in sequencing technologies have resulted in better molecular characterization and genotype-phenotype correlation of IRDs. This has fueled research into therapeutic development over the recent years. Animal models are required for pre-clinical efficacy assessment. Non-human primates (NHP) are ideal due to the anatomical and genetic similarities shared with humans. However, developing NHP disease to recapitulate the disease phenotype for specific IRDs may be challenging from both technical and cost perspectives. This review discusses the currently available NHP IRD models and the methods used for development, with a particular focus on gene-editing technologies.

Keywords: hereditary eye diseases; leber congenital amaurosis; retinitis pigmentosa; stargardt disease.

Publication types

  • Review

MeSH terms

  • Animals
  • Gene Editing
  • Phenotype
  • Primates / genetics
  • Retina
  • Retinal Diseases* / genetics
  • Retinal Diseases* / therapy