Developing Preclinical Models That Recapitulate Disease Phenotypes of Retinal Pigment Epithelium & Photoreceptor Cells for Efficient Translation Into the Clinic
Time: 9:10 am
day: Workshop Day
Details:
The lack of reliable preclinical models for dry AMD and geographic atrophy have proved to be a major hurdle in current AMD drug discovery and research. Several genetically modified mouse models previously developed have failed to fully recapitulate the AMD phenotypes, therefore, posing as a challenge during translation into the clinic.
Join this workshop to:
- Discuss the lack of accurate preclinical models replicating the retinal microenvironment including cell atrophy, loss of photoreceptor and RPE cells, deposition of drusen and the accumulation of lipofuscin, as seen in patients with dry AMD and GA
- Develop in vitro cell culture models (such as iPSC-derived RPE cell lines), organ-onchips and 3D organoids providing physiologically relevant models combining the various retinal cell types
- Overcome the lack of accurate in vivo models capturing different disease manifestations with the development of novel RPE-specific knockouts mouse models
- Uncover the latest advancements in tissue characterization techniques, including single-cell sequencing, spatial histology, proteomics, and RNA sequencing, and how these techniques provide insights into the origins of diseases by analyzing human tissues at a granular level
- Examine the challenges of comparing data across different models (oxidative stress vs genetically modified), emphasizing the importance of standardized methodologies to facilitate collaboration and data interpretation
