@article{e59598bbf02c410ca12da5ce7edf97b9,
title = "Intravenous administration of blood–brain barrier-crossing conjugates facilitate biomacromolecule transport into central nervous system",
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.",
author = "Chang Wang and Siyu Wang and Yonger Xue and Yichen Zhong and Haoyuan Li and Xucheng Hou and Kang, {Diana D.} and Zhengwei Liu and Meng Tian and Leiming Wang and Dinglingge Cao and Yang Yu and Jayce Liu and Xiaolin Cheng and Tamara Markovic and Alice Hashemi and Kopell, {Brian H.} and Charney, {Alexander W.} and Nestler, {Eric J.} and Yizhou Dong",
note = "Publisher Copyright: {\textcopyright} The Author(s), under exclusive licence to Springer Nature America, Inc. 2024.",
year = "2024",
doi = "10.1038/s41587-024-02487-7",
language = "English",
journal = "Nature Biotechnology",
issn = "1087-0156",
publisher = "Nature Research",
}