The blood-brain barrier is a huge challenge when it comes to the delivery of therapeutic proteins to treat genetic diseases, injury, and neurodegenerative diseases.
Ion channel dysfunctions lead to a wide array of illnesses including epilepsy, cardiac arrhythmia and type II diabetes. However, the number of clinically approved drugs for restoring normal ion channel function is limited.
Stanford researchers in the laboratory of Dr. Daria Mochly-Rosen have developed novel small molecules for modulating ALDH2 (mitochondrial aldehyde dehydrogenase-2).
Stanford researchers developed a first-in-class small-molecule inhibitor of the CLC-2 ion channel for research and drug development. CLC-2 is part of the CLC family of chloride ion channels, which regulate the flux of chloride ions across cell membranes.
Stanford and Rockefeller researchers have identified and developed dynein-specific inhibitors that have significant medical applications involving mitotic spindle assembly, organelle transport, and primary cilia formation.