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.

Original languageEnglish
JournalNature Biotechnology
DOIs
StateAccepted/In press - 2024

Fingerprint

Dive into the research topics of 'Intravenous administration of blood–brain barrier-crossing conjugates facilitate biomacromolecule transport into central nervous system'. Together they form a unique fingerprint.

Cite this