Related Experiment Video
Updated: Oct 5, 2025
09:00
Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
Published on: August 2, 2018
8.3K
The Role of Recombinant AAV in Precise Genome Editing.
Swati Bijlani1, Ka Ming Pang1, Venkatesh Sivanandam1
1Department of Surgery, Beckman Research Institute, City of Hope National Medical Center, Duarte, CA, United States.
Frontiers in Genome Editing
|January 31, 2022
Summary
Adeno-associated viruses (AAVs) offer precise genome editing via homologous recombination, avoiding off-target mutations. This novel gene therapy tool shows promise for correcting genetic diseases efficiently in vivo.
Area of Science:
- Molecular Biology
- Gene Therapy
- Virology
Background:
- Adeno-associated viruses (AAVs) are replication-defective, non-pathogenic viruses.
- Recombinant AAVs are established tools in genetic medicine and gene therapy.
- AAVs possess a unique ability for precise genome editing.
Purpose of the Study:
- To review the distinct properties of direct adeno-associated virus-mediated genome editing.
- To explore the potential mechanisms of action for AAV-mediated genome editing.
- To highlight AAVs as a promising platform for correcting disease-associated mutations.
Main Methods:
- Focus on the high-fidelity homologous recombination (HR) pathway utilized by AAVs.
- Discuss the absence of requirement for exogenous nucleases for DNA cleavage.
- Examine the efficiency and accuracy of stem cell-derived AAV (AAVHSC) editing in vitro and in vivo.
Main Results:
- AAVs enable precise genome editing through HR, without inducing indel mutations or off-target effects.
- AAVHSCs demonstrate efficient and accurate HR, even in post-mitotic cells and tissues.
- AAVs possess an intrinsic delivery mechanism, simplifying gene editing applications.
Conclusions:
- AAV-mediated genome editing is a highly precise and safe platform.
- This technology offers a promising approach for genetic disease correction without increasing mutational burden.
- AAVs represent a significant advancement in genome editing tools for therapeutic applications.
Related Concept Videos
02:53Conservative Site-specific Recombination and Phase Variation
6.2K
Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
The recognition sites for Cre recombinase called LoxP...
6.2K
01:28CRISPR/Cas9 Genome Editing
530
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...
530
01:59CRISPR
53.3K
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...
53.3K