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Updated: Jun 6, 2025
Intraventricular Drug Delivery and Sampling for Pharmacokinetics and Pharmacodynamics Study
Published on: March 31, 2022
Intravenous administration of blood-brain barrier-crossing conjugates facilitate biomacromolecule transport into
Chang Wang1,2,3,4,5,6,7, Siyu Wang1,2,3,4,5,6,7, Yonger Xue1,2,3,4,5,6,7
1Icahn Genomics Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Abstract:
Delivery of biomacromolecules to the central nervous system (CNS) remains challenging because of the restrictive nature of the blood-brain barrier (BBB). We developed a BBB-crossing conjugate (BCC) system that facilitates delivery into the CNS through γ-secretase-mediated transcytosis. Intravenous administration of a BCC10-oligonucleotide conjugate demonstrated effective transportation of the oligonucleotide across the BBB and gene silencing in wild-type mice, human brain tissues and an amyotrophic lateral sclerosis mouse model.
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