CRISPR: a tool with potential for genomic reprogramming in neurological disorders
Yogesh K Dhuriya1, Aijaz A Naik2
1Indian Institute of Toxicology Research (IITR), Lucknow, India.
Molecular Biology Reports
|December 12, 2022
Summary
CRISPR-Cas9 technology offers precise gene editing for neurological disorders by correcting mutations. This advanced RNA system overcomes limitations of older methods, paving the way for novel therapeutic strategies.
Area of Science:
- Neuroscience
- Genetics
- Molecular Biology
Background:
- Neurological disorders stem from disruptions in precise neural transcriptional programs during development.
- Lack of understanding of disease mechanisms hinders effective treatment development.
- CRISPR-Cas9 technology presents a novel approach for genetic correction.
Purpose of the Study:
- To discuss CRISPR-Cas9 technology, its mechanisms, and limitations.
- To explore CRISPR's potential in developing tools for neurological disorder treatment.
- To review current neuroscientific efforts in therapeutic gene correction.
Main Methods:
- Review of CRISPR-Cas9 technology and its RNA-guided mechanisms.
- Analysis of CRISPR-Cas9's precision in correcting disease-causing mutations.
- Comparison with RNA interference strategies and their limitations.
Main Results:
- CRISPR-Cas9 enables precise correction or silencing of mutations.
- This technology offers advantages over RNA interference by modulating gene expression.
- CRISPR-Cas9 holds significant promise for novel therapeutic applications.
Conclusions:
- CRISPR-Cas9 technology is a powerful tool for combating neurological disorders.
- Further research and application of CRISPR-Cas9 can lead to effective treatments.
- Neuroscientists are actively exploring CRISPR-based therapeutic corrections.
More Related Videos
09:39CRISPR-mediated Loss of Function Analysis in Cerebellar Granule Cells Using In Utero Electroporation-based Gene Transfer
Published on: June 9, 2018
9.9K
09:44Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases
Published on: May 2, 2025
294
Related Concept Videos
01:59CRISPR
52.7K
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...
52.7K
01:28CRISPR/Cas9 Genome Editing
130
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...
130
02:53Conservative Site-specific Recombination and Phase Variation
6.1K
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...
6.1K
02:31Homologous Recombination
50.8K
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...
50.8K