Shanghai Children's Medical Center Launches World's First Base Editing Clinical Study for Pediatric Chylomicronemia Syndrome
Professor Wang Xiumin's team at Shanghai Children's Medical Center led the launch of the world's first in vivo base editing drug clinical study for pediatric and adolescent familial chylomicronemia syndrome, exploring the safety and efficacy of LNP-delivered gene editing therapy. The hospital has built a registry of nearly 10,000 rare disease cases and conducts multiple cutting-edge studies including AADC deficiency gene therapy.
Shanghai Children's Medical Center
