Related Experiment Video
Updated: Aug 27, 2025
09:04
Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
Published on: September 21, 2017
9.6K
Nonviral delivery systems for antisense oligonucleotide therapeutics
Si Huang1,2,3, Xin-Yan Hao1,2,3, Yong-Jiang Li1,2,3
1Department of Pharmacy, the Second Xiangya Hospital, Central South University, Changsha, 410011, People's Republic of China.
Biomaterials Research
|September 30, 2022
Summary
Antisense oligonucleotides (ASOs) require effective delivery systems. Nonviral nanoparticles offer versatile, safe, and efficient options for ASO gene delivery, crucial for advancing genetic disorder treatments.
Area of Science:
- Biotechnology
- Genetic Medicine
- Nanotechnology
Background:
- Antisense oligonucleotides (ASOs) are vital for treating genetic disorders.
- Gene therapies, including ASOs, often necessitate vectors for protection and in vivo transport.
- Viral vectors have limitations, driving the need for alternative delivery methods.
Purpose of the Study:
- To review current nonviral vector strategies for antisense oligonucleotide (ASO) delivery.
- To highlight the importance of nonviral vectors in advancing ASO therapeutics.
- To discuss ASO modifications, mechanisms, and multi-carrier approaches for enhanced delivery.
Main Methods:
- Literature review of nonviral delivery strategies for ASOs.
- Analysis of ASO modifications and their impact on delivery.
- Examination of various nonviral vector platforms and their applications.
Main Results:
- Nonviral nanoparticles provide adaptable design and formulation for targeted ASO delivery.
- These vectors ensure protection against degradation and facilitate efficient transport in vivo.
- Successful applications in animal models and clinical settings demonstrate their therapeutic potential.
Conclusions:
- Nonviral vectors are essential for the progression of ASO-based therapies.
- Continued development of targeted nonviral delivery strategies will expand the therapeutic value of ASOs.
- Exploring nonviral vectors is inevitable with the advancement of new ASO drug development.
Related Concept Videos
02:30siRNA - Small Interfering RNAs
16.9K
Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
16.9K
01:23RNA Interference
26.3K
RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
26.3K
02:15Experimental RNAi
6.2K
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
6.2K