FKBPL Gene Therapy for Age-Related Macular Degeneration (360G-Wellcome-206765_Z_17_Z)

This research project is designed to interrogate an alternative therapeutic for age-related macular degeneration. FKBPL is an anti-angiogenic protein that has a mechanism of action independent of the VEGF pathway. As such this project will deliver DNA than encodes the FKBPL protein in vitro and the molecular markers of angiogenesis will be measured. The novel peptide delivery sequence, termed RALA (WO2014087023) will be used to deliver the FKBPL DNA to the nucleus of the cells where the therapeutic protein can be made. Key Goals include: Characterisation of the RALA/FKBPL nanoparticles In vitro culture of human retinal pigment epithelial cells (ARPE-19), ACBR 183 primary human retinal pericyte cells, ACBR181 primary human retinal microvascular endothelial cells and choroidal endothelial cells (RF6A) Learning a range of molecular techniques including FACS analysis, rtPCR, IHC, western blot and migration/tubule formation assays Development of transferable skills such as health and safety knowledge in a laboratory, presentation skills, data analysis, experimental design, critical thinking and scientific report writing skills. It is anticpated that at the end of this placement that the student will have acquired an in depth knowledge of what a career in research entails and have learnt several techniques that can be employed post degree.