Related Experiment Video
Updated: Jul 14, 2025
08:31
CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation
Published on: June 20, 2019
14.1K
Delivery of gene editing therapeutics
Bhavesh D Kevadiya1, Farhana Islam2, Pallavi Deol3
1Department of Pharmacology and Experimental Neuroscience, College of Medicine, University of Nebraska Medical Center, Omaha, NE 68198-5880, USA.
Nanomedicine : Nanotechnology, Biology, and Medicine
|October 9, 2023
Summary
Gene editing, including CRISPR-Cas9, offers potential for treating genetic and infectious diseases. Researchers are exploring safe delivery methods like lipid nanoparticles to overcome challenges with viral vectors.
Area of Science:
- Biotechnology
- Genetics
- Molecular Biology
Background:
- Gene editing technologies show promise for addressing genetic, infectious, immune, cancerous, and degenerative diseases.
- CRISPR-Cas9 gene editing is effective for modifying DNA to prevent or treat diseases.
Purpose of the Study:
- To review available gene delivery systems for treating and preventing a wide range of diseases.
- To highlight the potential of CRISPR-Cas9 gene editing in disease management.
Main Methods:
- Review of viral vector-based systems and nonviral vector lipid nanoparticles for gene delivery.
- Analysis of CRISPR-Cas9 delivery strategies and their associated immune responses.
Main Results:
- Viral vectors have high efficiency but limitations in packaging capacity and potential immune responses.
- Nonviral lipid nanoparticles have been successfully used as vaccine and therapeutic deliverables.
Conclusions:
- Safe and efficient delivery of CRISPR-Cas9 gene editing tools is crucial for therapeutic applications.
- Gene editing holds significant potential to revolutionize the treatment and prevention of chronic debilitating disorders.
Related Concept Videos
00:59Gene Therapy
25.5K
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...
25.5K
01:59CRISPR
52.1K
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...
52.1K
00:49What is Genetic Engineering?
74.3K
Overview
74.3K
01:29Microorganisms in Medicine and Therapeutics
37
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.
37
01:28CRISPR/Cas9 Genome Editing
27
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...
27