Intravenous administration of blood-brain barrier-crossing conjugates facilitate biomacromolecule transport into central nervous system.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 39587229.
- Also identified by DOI 10.1038/s41587-024-02487-7.
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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.
Medical subject headings
- Blood-Brain Barrier
- Central Nervous System
- Oligonucleotides