Evaluating the effects of aging on distribution and gene silencing activity of lipid-siRNA conjugates delivered into cerebrospinal fluid
1. 当前求助状态已完结, 请及时下载应助文件
2. 系统将在 2026-10-20 17:43:47 删除文件
注: 所有应助的资源仅供学习交流使用, 不得违反相关法律法规
DOI:
文献链接:
其他信息:
AP Ligocki, AG Sorets, AM Abdulrahman, N Francini…
Molecular Therapy, 2026
cell.com
Aging is the primary risk factor for chronic neurodegenerative diseases and is associated with alterations to cerebrospinal fluid (CSF) flow and clearance. CSF delivery is currently the most clinically advanced route of administration for oligonucleotide therapeutics, but it remains poorly understood how aging, which is rarely incorporated into clinical trials, impacts biodistribution, gene silencing activity, and potential toxicity of these compounds. Here, we evaluated a lipid-siRNA conjugate (L2-siRNA) for potential age-related changes to …
