生物通17 天
双基因替代疗法为枫糖尿病带来新希望：突破传统治疗局限
枫糖尿病（MSUD）通常是由编码支链 α- 酮酸脱氢酶 E1α 亚基（BCKDHA）或 E1β 亚基（BCKDHB）的基因突变引起的。目前，其治疗手段仅有严格控制饮食或异体肝移植。 在本研究中，研究人员设计了一种由双向启动子驱动、包含 BCKDHA 和 BCKDHB 的单基因替代构建体 ...
GEN - Genetic Engineering and Biotechnology News12 天
AAV Gene Therapy for Maple Syrup Urine Disease Shows Promise
A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.
Gene therapy for maple syrup urine disease shows promise
New gene therapy for maple syrup urine disease shows promise in preventing deadly symptoms in cows and offers hope for patients.