Investigating the loss of Complement Factor D on Complement Activity in the Stargardt Disease Mouse Model


2023-03-20, 2023-03-20


Griffith, Kaitlin

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Stargardt disease is the most common form of macular degeneration affecting children and young adults (Fujinami et al., 2015). This condition is, in part, caused by the dysregulation of the complement system leading to inflammation in tissue that is critical for vision. Complement Factor D (Cfd) is the rate limiting enzyme in the alternative complement pathway. We hypothesize that the loss of Cfd in the Stargardt disease mouse model will rescue the phenotype and reduce the pathological inflammation. This project investigates complement activity by developing a western blot assay to measure inactive (C3) and active (C3b) forms of complement to determine whether the loss of Cfd reduces complement activity. If the loss of Cfd rescues the phenotype observed in Stargardt model mice, this would support Cfd as a target for inhibition through pharmaceutical or gene therapy.



western blot, Stargardt disease, macular degeneration, Biology