Conditional expression of therapeutic cargo in neurons of autism spectrum disorders
Yugang Zhang received B.S. from College of Chemistry and Molecular Engineering in Peking University in 2014. In 2021, he completed Ph.D. in Cornell university in Department of Chemistry under the supervision of Dr. Hening Lin. In his doctoral work, he studied the biosynthesis and biological function of post-translational modifications on eukaryotic elongation factors.
Gene therapy requires delivery of nucleic acid polymers or ribonucleoproteins (RNPs) into human cells. However, current strategy is poor in delivery to targeted cell population. Cell type specific delivery will enhance the efficiency and reduce the side effects of gene therapy. Delivery of a conditional expression system, i.e., mRNA cargo that only turns on expression in the presence of certain RNA species, can achieve targeted delivery of cargos, as cell types can be defined by its transcriptome. By using Cas13 to target RNA, combining trans-splicing group I introns or collateral activity of Cas13, a conditional expression system can be designed. With the properly built delivery system to human neurons, my goal is to achieve the targeted gene therapy in human neurons for autism spectrum disorders.
Delivery, Gene Therapy, Targeted Therapy