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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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Literature Corpus work
98e6d2b3-57de-58fc-aecd-ac1d34cdbcaf
DOI
10.1101/2024.06.09.598079
Open publication

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Lipid-siRNA conjugate accesses a perivascular transport mechanism and achieves widespread and durable knockdown in the central nervous systemDOI 10.1101/2024.06.09.598079
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