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Related Experiment Video

Updated: Feb 8, 2026

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
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Self-Assembled Double-Bundle DNA Tetrahedron for Efficient Antisense Delivery.

Juanjuan Yang1, Qiao Jiang2, Lin He1

  • 1Bio-X Institutes, Key Laboratory for the Genetics of Developmental and Neuropsychiatric Disorders (Ministry of Education) , Shanghai Jiao Tong University , Shanghai 200240 , China.

ACS Applied Materials & Interfaces
|July 3, 2018
PubMed
Summary

This study presents a novel DNA nanocarrier for targeted drug delivery. The DNA tetrahedron efficiently delivers antisense oligonucleotides to silence the c-raf gene, inhibiting cancer cell proliferation.

Keywords:
antisense oligonucleotidescancer therapydouble-bundle DNA tetrahedrondrug deliveryself-assembly

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Area of Science:

  • Biomaterials Science
  • Molecular Biology
  • Nanotechnology

Background:

  • DNA nanostructures offer precise arrangement of functional components.
  • Antisense oligonucleotides hold therapeutic potential but face delivery challenges.

Purpose of the Study:

  • To design a DNA nanocarrier for efficient delivery of antisense oligonucleotides targeting the c-raf gene.
  • To investigate the intracellular delivery and therapeutic efficacy of the nanocarrier.

Main Methods:

  • Rational design of a double-bundle DNA tetrahedron.
  • Integration of antisense oligonucleotides and nuclear targeting peptides.
  • Cellular uptake studies and gene silencing assays in A549 cells.

Main Results:

  • The DNA tetrahedron successfully delivered antisense oligonucleotides into A549 cells.
  • Targeted delivery to the nucleus enhanced mRNA downregulation.
  • Antisense release in response to the intracellular environment inhibited cell proliferation without transfection reagents.
  • Efficient knockdown of the c-raf gene was achieved.

Conclusions:

  • A designer DNA nanocarrier system demonstrates efficient delivery of nucleic acid drugs.
  • This approach offers a promising new avenue for cancer therapy and gene silencing applications.