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Updated: Jan 20, 2026
07:47
Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
Published on: October 29, 2019
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Tissue-Specific Delivery of Oligonucleotides
Xin Xia1, Nicolette Pollock1, Jiehua Zhou1
1Department of Molecular and Cellular Biology, Beckman Research Institute, City of Hope, Duarte, CA, USA.
Methods in Molecular Biology (Clifton, N.J.)
|August 15, 2019
Summary
Oligonucleotides are key for gene therapies, but cellular delivery remains a challenge. Researchers are developing advanced delivery vehicles, including viral and nonviral systems, to improve their therapeutic potential for diseases like cancer.
Area of Science:
- Biotechnology
- Molecular Biology
- Therapeutic Drug Delivery
Background:
- Oligonucleotides, including short-interfering RNA (siRNA) and antisense oligonucleotides, are crucial for gene knockdown and editing.
- Their therapeutic application is significantly hindered by challenges in cellular delivery.
- Current gene therapy strategies leverage oligonucleotides for targeted gene modulation.
Purpose of the Study:
- To discuss the biological barriers affecting oligonucleotide delivery.
- To review recent advancements in tissue-specific oligonucleotide delivery systems.
- To explore the potential of viral and nonviral vectors in enhancing oligonucleotide therapeutics.
Main Methods:
- Review of existing literature on oligonucleotide delivery systems.
- Discussion of challenges posed by biological barriers.
- Analysis of viral and nonviral delivery vectors for oligonucleotide therapeutics.
Main Results:
- Cellular delivery of oligonucleotides is a major obstacle for their therapeutic use.
- Various delivery vehicles, including viral vectors, nanoparticles, liposomes, and polymers, are being explored.
- Tissue-specific delivery is achievable through careful design of these delivery systems.
Conclusions:
- Overcoming cellular delivery barriers is essential for realizing the full therapeutic potential of oligonucleotides.
- Advanced delivery systems, both viral and nonviral, show promise for targeted gene therapies.
- Judicious design and formulation of delivery vehicles can significantly improve oligonucleotide efficacy and expand their therapeutic applications.
Keywords:
AntibodiesAptamersEPREndosomal escapeLigandsOligonucleotide therapeuticsPhagocytosisTissue-specificMore Related Videos
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