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

Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.

You might also read

Related Articles

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

Sort by
Same author

Combining Anti-Inflammatory Drugs and Cell-Specific Expression-Suppression Reduces the Adjuvant Activity of mRNA-LNPs.

Molecular pharmaceutics·2026
Same author

Lipid nanoparticle-encapsulated microRNA-192: An anti-inflammatory adjuvant that enhances vaccine efficacy in aged mice.

Molecular therapy. Nucleic acids·2025
Same author

Lipid nanoparticle-mediated mRNA delivery system into preimplantation embryos†.

Biology of reproduction·2025
Same author

Combination of ionizable lipids with oleic acid and vitamin E scaffolds for RNA cancer vaccine delivery.

Journal of controlled release : official journal of the Controlled Release Society·2025
Same author

Neoantigen mRNA vaccines induce progenitor-exhausted T cells that support anti-PD-1 therapy in gastric cancer with peritoneal metastasis.

Gastric cancer : official journal of the International Gastric Cancer Association and the Japanese Gastric Cancer Association·2025
Same author

Modulating Immunogenicity and Reactogenicity in mRNA-Lipid Nanoparticle Vaccines through Lipid Component Optimization.

ACS nano·2025

Related Experiment Video

Updated: Jul 6, 2026

Expression of Fluorescent Fusion Proteins in Murine Bone Marrow-derived Dendritic Cells and Macrophages
09:07

Expression of Fluorescent Fusion Proteins in Murine Bone Marrow-derived Dendritic Cells and Macrophages

Published on: October 30, 2018

Nonviral gene delivery.

Hidetaka Akita1, Hideyoshi Harashima

  • 1Laboratory for Molecular Design of Pharmaceutics, Faculty of Pharmaceutical Sciences, Hokkaido University, Sapporo, and CREST Japan Science and Technology Agency, Saitama, Japan.

Contributions to Nephrology
|April 9, 2008
PubMed
Summary
This summary is machine-generated.

Nonviral gene therapy for renal failure faces challenges with low delivery efficiency. Novel

More Related Videos

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts
11:11

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Published on: December 17, 2016

Related Experiment Videos

Last Updated: Jul 6, 2026

Expression of Fluorescent Fusion Proteins in Murine Bone Marrow-derived Dendritic Cells and Macrophages
09:07

Expression of Fluorescent Fusion Proteins in Murine Bone Marrow-derived Dendritic Cells and Macrophages

Published on: October 30, 2018

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts
11:11

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Published on: December 17, 2016

Area of Science:

  • Biomedical Engineering
  • Molecular Biology
  • Renal Medicine

Background:

  • Gene and RNA interference therapies offer potential treatments for intractable renal failure.
  • A major hurdle in nonviral gene therapy is the inefficient delivery of therapeutic nucleic acids into target cell nuclei.
  • Current methods like mechanical techniques and topical preparations have limitations in controlling intracellular trafficking.

Purpose of the Study:

  • To address the limitations of current nonviral gene delivery systems for renal failure.
  • To explore the development of novel vectors based on the 'programmed packaging' concept for enhanced intracellular trafficking.
  • To establish methods for quantitative evaluation of intracellular trafficking for engineered gene vectors.

Main Methods:

  • Review of existing mechanical and topical delivery techniques for renal gene therapy.
  • Conceptualization of 'programmed packaging' vectors integrating multiple functions.
  • Emphasis on quantitative evaluation and comparison of intracellular trafficking with viral vectors like adenovirus.

Main Results:

  • Identified low delivery efficiency as a key obstacle in nonviral gene therapy for renal failure.
  • Highlighted the need for improved control over intracellular trafficking for enhanced efficacy.
  • Proposed 'programmed packaging' as a strategy for developing advanced gene vectors.

Conclusions:

  • Novel vectors with integrated functions are desirable for overcoming current gene delivery challenges.
  • Quantitative assessment of intracellular trafficking is crucial for evaluating and optimizing gene vector efficacy.
  • Comparing engineered vectors with viral systems aids in identifying rate-limiting steps in intracellular transport.