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Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood-brain barrier and knock down genes in the rodent CNS.

Author: ArimuraNaoto, BennettC Frank, DwyerChrissa A, EbiharaSatoe, HaraRintaro Iwata, HirabayashiHideki, IgariTomoko, IharaKensuke, IshiiTakashi, KaburagiHidetoshi, MiyataHaruka, MiyataKanjiro, MiyataKenichi, NagataTetsuya, OhyagiMasaki, PowersBerit, RigoFrank, SethPunit P, UchiharaToshiki, WadaTakeshi, YamamotoSyunsuke, YokotaTakanori, Yoshida-TanakaKie, YoshiokaKotaro

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

This study demonstrates the potential of DNA/RNA heteroduplex oligonucleotides (HDOs) for targeted gene knockdown in the central nervous system (CNS). Cholesterol-conjugated HDOs effectively cross the blood-brain barrier, achieving significant gene suppression in major CNS cell types without requiring intrathecal administration. The findings offer a promising approach for developing ASO-based therapies for neurological diseases.
Paper Details　
Original Abstract of the Article :
Achieving regulation of endogenous gene expression in the central nervous system (CNS) with antisense oligonucleotides (ASOs) administered systemically would facilitate the development of ASO-based therapies for neurological diseases. We demonstrate that DNA/RNA heteroduplex oligonucleotides (HDOs) ...See full text at original site
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引用元：
https://doi.org/10.1038/s41587-021-00972-x

データ提供：米国国立医学図書館（NLM）

Cholesterol-Functionalized DNA/RNA Heteroduplexes: A New Path Through the Blood-Brain Barrier Desert

The blood-brain barrier, a protective shield surrounding the central nervous system (CNS), is a formidable obstacle for many medications. This research, like a group of scientists seeking a new route through a desert, explores the potential of cholesterol-functionalized DNA/RNA heteroduplex oligonucleotides (HDOs) to deliver therapeutic molecules to the CNS. The researchers sought to develop a more effective method for targeting genes in the brain and treating neurological diseases.

HDOs: A New Oasis in the Blood-Brain Barrier Desert?

The research demonstrated that cholesterol-conjugated HDOs, like a camel caravan equipped with a special map, can successfully cross the blood-brain barrier after subcutaneous or intravenous administration in mice and rats. These HDOs, like a refreshing oasis in the desert, distributed throughout the brain, spinal cord, and peripheral tissues, effectively suppressing the expression of target genes in the CNS. This research suggests that HDOs may offer a promising alternative to traditional ASOs for treating neurological disorders.

Navigating the CNS: A Desert Oasis of Knowledge

This study provides exciting new possibilities for developing therapeutic approaches to neurological diseases. The ability to effectively deliver therapeutic molecules to the CNS, like finding a hidden oasis in the desert, opens up new avenues for research and treatment development. Further research is needed to optimize the delivery and efficacy of HDOs, paving the way for innovative therapies to treat these challenging conditions.

Dr.Camel's Conclusion

The desert of neurological diseases can be a difficult landscape to navigate, but this research offers a glimmer of hope for patients seeking new treatments. Cholesterol-functionalized HDOs, like a well-equipped camel caravan, can traverse the blood-brain barrier, delivering therapeutic molecules to the brain and potentially offering new hope for treating neurological disorders. The journey towards effective treatments continues, but this research offers a valuable new path for exploration.

Date :
  1. Date Completed 2022-04-21
  2. Date Revised 2023-02-06
Further Info :

Pubmed ID

34385691

DOI: Digital Object Identifier

10.1038/s41587-021-00972-x

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Languages

English

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