The overarching goal of Dr. Lu’s research is to combine genetics and pharmacology to develop neural circuit-selective therapies for neuropsychiatric disorders. Trained as a psychiatrist and neuropharmacologist, Dr. Lu completed his postdoctoral training in molecular genetics and was appointed as an Assistant Researcher at Center for Neurobehavioral Genetics of the University of California, Los Angeles (UCLA). Dr. Lu developed the first BAC transgenic Parkinson’s disease (PD) mouse model that recapitulates the cardinal features of PD. His translational study of Huntington’s disease (HD) identified a novel therapeutic strategy (Sci. Transl. Med., 2015; Highlighted in Nat Rev Drug Discov. and was selected as the most influential paper of 2015 by HD insight). Dr. Lu co-invented a single-cell transgenic technology (MORF, Mosaicism with Repeat Frameshift) that received support from the first round of the Brain Initiative award. Funded by a NARSAD Young Investigator Award, Lu lab generated the next-generation mouse model for schizophrenia (Mol Psychiatry, 2019). Recently, Dr. Lu’s lab invented a robust method for efficient and precise CRISPR/Cas9 mediated genome editing in the adult mammalian brain via intravascular administration of a neurotropic AAV. His team is now actively pursuing therapeutic genome editing for devastating brain disorders, such as Down syndrome .
PI: Xiao-Hong Lu, M.D., Ph.D.