Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

18.8K
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...
18.8K
Nucleic Acid Structure01:25

Nucleic Acid Structure

9.7K
The pentose sugar in DNA is deoxyribose, while in RNA the pentose sugar is ribose. The difference between the sugars is the presence of the hydroxyl group on the ribose's second carbon and a hydrogen on the deoxyribose's second carbon. The phosphate residue attaches to the hydroxyl group of the 5′ carbon of one sugar and the hydroxyl group of the 3′ carbon of the sugar of the next nucleotide, which forms  a 5′ to 3′ phosphodiester linkage.
DNA Structure
DNA...
9.7K

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Exploring the impact of nucleotide length on lipid nanoparticle structure and properties.

International journal of pharmaceutics·2026
Same author

Discovery and Optimization of a Potent, Efficacious, and Brain-Penetrant Inhibitor of KRAS G12C.

Journal of medicinal chemistry·2026
Same author

Lipid Nanoparticle Development in Practice: Challenges and Collective Insights.

Pharmaceutical research·2026
Same author

Aminophosphonate-Derived Lipid Nanoparticles Enable Circular RNA Delivery for Functional Recovery after Spinal Cord Injury.

Materials today (Kidlington, England)·2025
Same author

The Future of Pharmaceutics: Showcasing Emerging Leaders in Drug Delivery.

Molecular pharmaceutics·2024
Same author

Discovery of potent dihydro-oxazinoquinolinone inhibitors of GuaB for the treatment of tuberculosis.

Bioorganic & medicinal chemistry letters·2024

Related Experiment Video

Updated: Feb 28, 2026

Formulating and Characterizing Lipid Nanoparticles for Gene Delivery using a Microfluidic Mixing Platform
09:41

Formulating and Characterizing Lipid Nanoparticles for Gene Delivery using a Microfluidic Mixing Platform

Published on: February 25, 2021

25.6K

Unconventional Lysine-Type Lipid Assemblies Enable Efficient Antisense Oligonucleotide Delivery with Distinct

Jieyan He1,2, Whitney Shatz-Binder3, Alexandra Robles3

  • 1Biochemical and Cellular Pharmacology, Genentech, 1 DNA Way, South San Francisco, CA 94080, USA.

Pharmaceutics
|February 27, 2026
PubMed
Summary

Researchers explored novel lipid formulations for delivering antisense oligonucleotides (ASOs). Lysine-type lipoplexes showed promising ASO delivery efficiency and stability, suggesting potential for genetic and neurological disorder treatments.

Keywords:
ASOLNPcationic lipidslipoplexnon-viral delivery systemstructure

More Related Videos

Assembly and Characterization of Polyelectrolyte Complex Micelles
08:44

Assembly and Characterization of Polyelectrolyte Complex Micelles

Published on: March 2, 2020

11.6K
Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
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

10.0K

Related Experiment Videos

Last Updated: Feb 28, 2026

Formulating and Characterizing Lipid Nanoparticles for Gene Delivery using a Microfluidic Mixing Platform
09:41

Formulating and Characterizing Lipid Nanoparticles for Gene Delivery using a Microfluidic Mixing Platform

Published on: February 25, 2021

25.6K
Assembly and Characterization of Polyelectrolyte Complex Micelles
08:44

Assembly and Characterization of Polyelectrolyte Complex Micelles

Published on: March 2, 2020

11.6K
Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
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

10.0K

Area of Science:

  • Biotechnology
  • Drug Delivery Systems
  • Molecular Biology

Background:

  • Antisense oligonucleotides (ASOs) offer therapeutic potential for genetic and neurological disorders by modulating gene expression.
  • Effective delivery of ASOs remains a significant challenge, with current research primarily focusing on lipid nanoparticles (LNPs).
  • Alternative formulations, preparation methods, and lipid compositions for optimizing ASO delivery require further exploration.

Purpose of the Study:

  • To investigate the potential of lysine-type cationic lipids in formulating lipoplexes (LPXes) and LNPs for enhanced ASO delivery.
  • To compare the formulation stability, cellular entry mechanisms, ASO delivery efficiency, and immune responses of novel lipid formulations against a benchmark.

Main Methods:

  • Screening of a lysine-type lipid mini-library to identify stable and safe cationic lipids (K3C14, K3C16).
  • Characterization of LPX and LNP formulations' physicochemical properties, cytotoxicity, ASO delivery efficiency, and immunogenicity.
  • Structural analysis of novel lipid assemblies using cryogenic electron microscopy (Cryo-EM).

Main Results:

  • Lysine-type lipids with K3 spacer or C14 fatty acid tails demonstrated excellent stability and safety.
  • The K3C16 lipoplex formulation exhibited ASO delivery efficiency and immune responses comparable to the SpikeVax LNP benchmark.
  • Cryo-EM revealed unique structures: K3C14 lipoplexes formed rouleaux-like structures, and K3C16 lipoplexes formed lipid nanosheet-like structures.

Conclusions:

  • Novel lipid assemblies, specifically lysine-type lipoplexes, show significant potential for efficient ASO delivery.
  • Unconventional lipid structures may offer alternative strategies for overcoming ASO delivery challenges.
  • These findings open new avenues for developing advanced ASO-based therapeutics.