Project:	Investigating Age-Related Macular Degeneration Using a "Disease in a Dish" Model
Disciplines:	Biology, Medical Research
Mentor:	Deborah Ferrington, Professsor- UCLA, (BBE), dferrington@doheny.org, Phone: 323-342-6404
AO Contact:	Dr. Peng Shang, pshang@doheny.org
Background:	This project is being offered by a UCLA Professor and is open only to Caltech students. The project will be conducted at the UCLA-affiliated Doheny Eye Institute at 150 N. Orange Grove Ave in Pasadena. Doheny Scientists conduct research relevant to ophthalmological diseases and retinal biology. The Ferrington Laboratory specializes in studying age-related macular degeneration (AMD), the leading cause of blindness in the elderly. Our team explores key questions in AMD research: What cellular changes occur with aging? What factors “tip the balance” to pathology? How do the cells respond to disease, and how can we mitigate these pathologic changes? To address these questions, we utilize induced pluripotent stem cells (iPSC) derived from AMD patients. These iPSCs are differentiated into retinal cells (iPSC-RPE) to study disease pathology and evaluate potential drug therapies. Both genetic and environmental risk factors contribute to the development and progression of AMD. The genetic risk we focus on is a prevalent single-nucleotide polymorphism (SNP) located in the gene encoding complement factor H (CFH). The environment risk factor we test in our experimental system is smoking, which significantly increases the risk for developing AMD. We have developed an in vitro model using iPSC-RPE cells derived from AMD patients that carry the low-risk or high-risk (Y402H) SNP in CFH. Using cigarette smoke extract to perturb the system, we study how the presence of the CFH risk SNP influences the response of iPSC-RPE cells to chronic smoke exposure.
Description:	SURF students will engage with lab scientists to learn a variety of molecular and biochemical assays that are appropriate for their project. Examples include culturing retinal cells and testing their response using assays, such as western blot, enzyme-linked immunosorbent assay (ELISA), immunostaining, and real-time polymerase chain reaction (PCR), to measure the protein or mRNA levels of genes of interest. Students may also have the opportunity to perform bioinformatic analysis of large proteomic and metabolomic data sets that were previously generated from the iPSC-RPE cells.
References:	https://doheny.org/research/ferrington/
Student Requirements:	Background in cell biology, molecular biology, biochemistry. Familiarity with data analysis in biological sciences is beneficial but not mandatory.
Programs:	This AO can be done under the following programs:   Program    Available To        SURF    Caltech students only  Click on a program name for program info and application requirements.