Related Experiment Video
Updated: Jan 1, 2026
08:20
A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
Published on: September 2, 2021
4.5K
Strategies for the CRISPR-Based Therapeutics.
Bin Li1, Yuyu Niu2, Weizhi Ji2
1Department of Infectious Disease, Shenzhen People's Hospital, The Second Clinical Medical College of Jinan University, The First Affiliated Hospital of Southern University of Science and Technology, Shenzhen 518020, China.
Trends in Pharmacological Sciences
|December 22, 2019
Summary
CRISPR genome editing technology, a microbial defense system, is now a powerful tool for genetic engineering. Researchers are improving its delivery and regulation for therapeutic applications in clinical trials.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Background:
- CRISPR-Cas is an RNA-guided nuclease system derived from microbial adaptive immunity.
- It has been repurposed as a versatile genome editing tool for eukaryotic systems.
- Significant efforts are focused on enhancing its efficiency and delivery methods.
Purpose of the Study:
- To review strategies for regulating and delivering CRISPR-Cas components.
- To outline current therapeutic applications and clinical translation of CRISPR-Cas technology.
Main Methods:
- Review of existing literature on CRISPR-Cas system improvements.
- Analysis of ex vivo and in vivo delivery strategies.
- Examination of preclinical and clinical trial data.
Main Results:
- Various methods have been developed to optimize CRISPR-Cas genome editing.
- Effective delivery systems are crucial for therapeutic applications.
- CRISPR-Cas is progressing towards clinical use in treating diseases.
Conclusions:
- CRISPR-Cas technology holds immense promise for genetic engineering and therapeutic interventions.
- Continued research into regulation and delivery will accelerate its clinical translation.
- The system's adaptability supports diverse applications in medicine.
More Related Videos
Related Concept Videos
01:59CRISPR
57.3K
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...
57.3K
01:28CRISPR/Cas9 Genome Editing
1.5K
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...
1.5K
02:53CRISPR and crRNAs
18.6K
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...
18.6K
02:31Homologous Recombination
62.1K
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...
62.1K
01:23The Antiviral System of Bacteria and Archaea: CRISPR
556
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats is a adaptive immune system found in bacteria and archaea that protects against viral infections. This system enables prokaryotic cells to identify, remember, and neutralize foreign genetic elements, primarily bacteriophages, by storing fragments of the invader’s DNA as a genetic memory.The CRISPR immune response begins during an initial infection. Cas (CRISPR-associated) proteins play a central role in this...
556
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