题目: Neurodegenerative Diseases: Genetic Causes, Pathogenic Mechanisms and CRISPR/Cas9-mediated Gene therapy.
报告人: Prof. Han-Xiang Deng
Research Professor of Neurology (Neuromuscular Disease)
时间: 2018年6月28日 星期四 13:00-15:00
Neurodegenerative Diseases are a group of disorders caused by neuronal dysfuînction or degeneration. The causes of these diseases are largely unknown. Approximately 5-10 % of the patients have a family history. These familial patients provide unique resources to identify genetic causes and to investigate the molecular pathways underlying the diseases. Our group has identified several disease-causing genes for Parkinson's disease, amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth (CMT) disease and other disorders. We have shown that oxidation-mediated protein aggregation, increased intracellular calcium concentration, and trafficking/recycling defects of synaptic vesicles and large dense-core vesicles underlie the pathogenic processes. Finally, I will discuss efficacy and safety issues related to CRISPR/Cas9-mediated in vivo specific gene-targeting as a therapeutic strategy for these diseases.
邀请人: 饶毅 教授