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

CRISPR01:59

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced Short...
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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.
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...

You might also read

Related Articles

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

Sort by
Same author

Design of novel interlocked bi-layer NiTi braided stent with ultra-thin walls for urinary tract obstruction treatment.

Regenerative biomaterials·2026
Same author

Identifying Risk-De-Escalating Markers in PREVENT-Defined Intermediate-Risk Older Adults: Insights From ASPREE.

Circulation. Population health and outcomes·2026
Same author

Directional Preparation of Duckweed-Based Porous Carbon and Adsorptive Removal of Volatile Organic Compounds.

ACS omega·2026
Same author

A lipidomic based metabolic age score for monitoring the effects of lifestyle and diet on metabolic disease risk.

Research square·2026
Same author

Toward precise management of groundwater by combined heavy metal(loid)s contamination at industrial sites: A machine learning driven source-to-risk zoning framework.

Environmental pollution (Barking, Essex : 1987)·2026
Same author

The role of Lipoprotein(a) and oxidized phospholipids in modifying the effects of aspirin on major cardiovascular events and bleeding in the ASPirin in Reducing Events in the Elderly (ASPREE) randomized clinical trial: Statistical analysis plan.

medRxiv : the preprint server for health sciences·2026

Related Experiment Video

Updated: Jul 15, 2026

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
09:51

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.

Meijia Yang1, Yiqiong Song1, Ziyang Wang2

  • 1National Engineering Laboratory For Internet Medical Systems and Applications The First Affiliated Hospital of Zhengzhou University Zhengzhou Henan China.

Medcomm
|July 14, 2026
PubMed
Summary

Advancements in genome editing tools like CRISPR are promising, but safe and effective delivery to target tissues remains a challenge. New viral and nonviral delivery systems are emerging to overcome these hurdles for therapeutic applications.

Keywords:
AAVsCRISPR–CasLNPsVLPsdelivery systemsgenome editing

More Related Videos

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
06:10

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates

Published on: May 9, 2025

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
07:56

Genome Editing in Mammalian Cell Lines using CRISPR-Cas

Published on: April 11, 2019

Related Experiment Videos

Last Updated: Jul 15, 2026

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
09:51

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
06:10

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates

Published on: May 9, 2025

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
07:56

Genome Editing in Mammalian Cell Lines using CRISPR-Cas

Published on: April 11, 2019

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Gene Therapy

Background:

  • Programmable nucleases (ZFNs, TALENs, CRISPR-based editors) have advanced therapeutic genome editing.
  • Clinical translation is hindered by challenges in safe, efficient, and tissue-specific delivery.

Purpose of the Study:

  • To provide a comprehensive overview of current and emerging delivery systems for therapeutic genome editing.
  • To highlight key innovations, challenges, and strategies for unlocking broader therapeutic potential.

Main Methods:

  • Review of viral vectors (e.g., adeno-associated viruses) and nonviral platforms (e.g., ionizable lipid nanoparticles).
  • Exploration of emerging modalities like nanosystems, extracellular vesicles, peptides, and scaffolds.
  • Discussion of high-throughput screening and machine learning for vector optimization.

Main Results:

  • Viral vectors offer durable editing but face limitations in cargo capacity and immunogenicity.
  • Nonviral platforms show high efficacy, particularly for hepatic targets.
  • Emerging modalities expand delivery options for transient expression and programmable targeting.

Conclusions:

  • Delivery systems are crucial for the clinical success of genome editing therapies.
  • Interdisciplinary strategies and technological advancements are key to overcoming current challenges.
  • Further innovation in delivery is poised to unlock the full therapeutic potential of genome editing.