Article
Lipid-siRNA conjugate accesses a perivascular transport mechanism and achieves widespread and durable knockdown in the central nervous system
2024-06-10
Abstract excerpt
Short-interfering RNA (siRNA) has gained significant interest for treatment of neurological diseases by providing the capacity to achieve sustained inhibition of nearly any gene target. Yet, efficacious drug delivery throughout deep brain structures of the CNS remains a considerable hurdle for intrathecally administered therapeutics. We herein describe an albumin-binding lipid-siRNA conjugate that transports along...
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Identifiers and source
- Literature Corpus work
- 98e6d2b3-57de-58fc-aecd-ac1d34cdbcaf
- DOI
- 10.1101/2024.06.09.598079
