Related Experiment Video
Updated: Dec 10, 2025
09:51
Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
Published on: May 25, 2018
35.1K
CRISPR-Cas9 DNA Base-Editing and Prime-Editing.
Ariel Kantor1,2, Michelle E McClements1,2, Robert E MacLaren1,2
1Nuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford OX3 9DU, UK.
International Journal of Molecular Sciences
|September 3, 2020
Summary
Base editing, a CRISPR-Cas technology, precisely modifies DNA without double-strand breaks. Adeno-associated vectors (AAV) show promise for delivering these advanced gene editing tools to treat genetic diseases.
Area of Science:
- Genetics and Molecular Biology
- Biotechnology
- Gene Therapy
Background:
- Genetic diseases stem from DNA base-pair alterations.
- CRISPR-Cas technologies have evolved into base editing, enabling precise point mutations without double-strand DNA breaks (DSBs).
- Cytosine base editors (CBEs) and adenine base editors (ABEs) are key types of DNA base editors.
Purpose of the Study:
- To review the development of various base editors.
- To assess the technical advantages and limitations of base editing technologies.
- To discuss the therapeutic potential of base editors for human diseases.
Main Methods:
- Review of CRISPR-Cas-based base editing technologies, including cytosine base editors (CBEs), adenine base editors (ABEs), and prime editing (PE).
- Evaluation of adeno-associated vectors (AAV) as a delivery platform for base editors.
- Analysis of the technical capabilities and challenges of current base editing systems.
Main Results:
- Base editing systems, including CBEs, ABEs, and PE, offer precise genomic DNA modifications.
- Prime editing (PE) expands the scope to all transition/transversion mutations and small insertions/deletions.
- Adeno-associated vectors (AAV) are identified as a leading viral delivery platform due to their favorable characteristics for base editor delivery.
Conclusions:
- Base editing represents a significant advancement in genome editing, offering precise genetic modifications.
- Efficient and safe delivery of base editors, particularly using AAV vectors, is crucial for therapeutic applications.
- Base editing technologies hold substantial therapeutic potential for treating a range of debilitating human genetic diseases.
Related Concept Videos
01:28CRISPR/Cas9 Genome Editing
1.3K
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...
1.3K
01:59CRISPR
56.5K
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...
56.5K
02:53CRISPR and crRNAs
18.4K
Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
18.4K
02:31Homologous Recombination
61.6K
The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
61.6K
02:53Conservative Site-specific Recombination and Phase Variation
6.5K
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...
6.5K