2. Academic Validation
  3. Intravenous administration of blood-brain barrier-crossing conjugates facilitate biomacromolecule transport into central nervous system

Intravenous administration of blood-brain barrier-crossing conjugates facilitate biomacromolecule transport into central nervous system

  • Nat Biotechnol. 2024 Nov 25. doi: 10.1038/s41587-024-02487-7.
Chang Wang # 1 2 3 4 5 6 7,  Siyu Wang # 1 2 3 4 5 6 7,  Yonger Xue 1 2 3 4 5 6 7,  Yichen Zhong 1 2 3 4 5 6 7,  Haoyuan Li 1 2 3 4 5 6 7,  Xucheng Hou 1 2 3 4 5 6 7,  Diana D Kang 1 2 3 4 5 6 7 8,  Zhengwei Liu 1 2 3 4 5 6 7,  Meng Tian 1 2 3 4 5 6 7,  Leiming Wang 1 2 3 4 5 6 7,  Dinglingge Cao 1 2 3 4 5 6 7,  Yang Yu 8,  Jayce Liu 8,  Xiaolin Cheng 8 9,  Tamara Markovic 6 10,  Alice Hashemi 11,  Brian H Kopell 11,  Alexander W Charney 11,  Eric J Nestler 12 13,  Yizhou Dong 14 15 16 17 18 19 20
Affiliations

Affiliations

  • 1 Icahn Genomics Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • 2 Marc and Jennifer Lipschultz Precision Immunology Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • 3 Department of Immunology and Immunotherapy, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • 4 Department of Oncological Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • 5 The Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • 6 Friedman Brain Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • 7 Biomedical Engineering and Imaging Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • 8 College of Pharmacy, The Ohio State University, Columbus, Columbus, OH, USA.
  • 9 Translational Data Analytics Institute, The Ohio State University, Columbus, OH, USA.
  • 10 Nash Family Department of Neuroscience, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • 11 Charles Bronfman Institute for Personalized Medicine, Department of Neurosurgery, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • 12 Friedman Brain Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. eric.nestler@mssm.edu.
  • 13 Nash Family Department of Neuroscience, Icahn School of Medicine at Mount Sinai, New York, NY, USA. eric.nestler@mssm.edu.
  • 14 Icahn Genomics Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. yizhou.dong@mssm.edu.
  • 15 Marc and Jennifer Lipschultz Precision Immunology Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. yizhou.dong@mssm.edu.
  • 16 Department of Immunology and Immunotherapy, Icahn School of Medicine at Mount Sinai, New York, NY, USA. yizhou.dong@mssm.edu.
  • 17 Department of Oncological Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA. yizhou.dong@mssm.edu.
  • 18 The Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. yizhou.dong@mssm.edu.
  • 19 Friedman Brain Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. yizhou.dong@mssm.edu.
  • 20 Biomedical Engineering and Imaging Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. yizhou.dong@mssm.edu.
  • # Contributed equally.
PMID: 39587229 DOI: 10.1038/s41587-024-02487-7
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.

Figures
Products
嗨！很高兴为您提供帮助！

尊敬的 MCE 客户您好，
请您选择所在区域，我们将转接对应客服为您服务！

热门区域

A

B

C

F

G

H

J

L

N

Q

S

T

X

Y

Z

热 A B C F G H J L N Q S T X Y Z