生物通5 天
腺相关病毒载体(AAV8/AAV9)在黏多糖贮积症IVA型小鼠模型中的组织特异 ...
本研究系统评估了AAV8/AAV9载体搭载不同启动子（TBG/LSPX肝特异性、CAG广谱性、LMTP肝肌串联、LBTP肝骨串联）对MPS IVA（Morquio A ...
EurekAlert!26 天
Prevalence of neutralizing antibodies to AAV2 and AAV9 in individuals with Niemann-Pick disease
Click here to read the article now. NPC1 is a rare, fatal neurodegenerative disorder, for which systemic AAV9-based gene therapy in mouse models has shown some success. The presence of ...
Nature4 年
AAV9-Based Gene Therapy in Monogenic Neurologic Diseases
A recombinant AAV9 capsid shell able to deliver payload across the blood-brain barrier and into the nervous system and many other tissues · Self-complementary (sc) AAV DNA technology, featuring ...
News Medical26 天
Many individuals with Niemann-Pick disease lack neutralizing antibodies against AAV2 or AAV9
NPC1 is a rare, fatal neurodegenerative disorder, for which systemic AAV9-based gene therapy in mouse models has shown some success. The presence of neutralizing antibodies against AAV can reduce ...
News Medical on MSN26 天
Many individuals with Niemann-Pick disease lack neutralizing antibodies against AAV2 or AAV9
The investigators found that more than half of patients were seropositive at both time points, with 68.2% of individuals ...