Research Summary
The Knight Initiative supports drug discovery and screening for potential therapies through laboratory research. We are partnering with the Stanford Innovative Medicines Accelerator (IMA) to develop this drug-discovery pipeline. The IMA is a Stanford initiative that seeks to accelerate the translation of Stanford research discoveries into new medicines while expanding our knowledge of human biology. Lysosomal dysfunction in certain neurodegenerative disorders, including Parkinson’s disease, is caused by alterations in the essential lysosomal lipid bis(monoacylglycero)phosphate (BMP), and leads to the death of neurons. The Abu-Remaileh lab has identified the elusive BMP synthase in humans. This project will enable the prototyping of a novel enzyme activator for selective restoration of BMP levels to slow neurodegeneration.