Abstract

SummaryDisease‐causing variants of a large number of genes trigger inherited retinal degeneration leading to photoreceptor loss. Because cones are essential for daylight and central vision such as reading, mobility and face recognition, the presentation will focus on a variety of animal models for cone diseases. The pertinence of using these models to reveal genotype/phenotype correlations and to evaluate new therapeutic strategies will bediscussed. Interestingly, several large animal models recapitulate human diseases and can serve as a strong base to study the disease biology and to assess the scale‐up of new therapies. Examples of innovative approaches will be presented such as lentiviral‐based transgenesis in pigs. The models we will discuss permit to explore common mechanisms existing between different species and to highlight the pathways that may be specific to primates, including human.

Full Text
Paper version not known

Talk to us

Join us for a 30 min session where you can share your feedback and ask us any queries you have

Schedule a call

Disclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.