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Related Concept Videos

CRISPR01:59

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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...
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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.
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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...
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Recent progress in CRISPR-Cas-system for neurological disorders.

Sarita Mishra1, Shuvomoy Banerjee2, Budhi Sagar Tiwari3

  • 1National Forensic Sciences University, Gandhinagar, Gujarat, India.

Progress in Molecular Biology and Translational Science
|January 17, 2025
PubMed
Summary

CRISPR-Cas gene editing offers a promising new approach for treating neurodegenerative diseases like Parkinson's and Alzheimer's by precisely targeting genetic mutations. This technology enables the development of novel gene therapies for these complex neurological conditions.

Keywords:
CRISPR-Cas systemGene editingGene therapyNervous systemNeurological disordersNeurons

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Area of Science:

  • Neuroscience
  • Genetics
  • Biotechnology

Background:

  • Neurological diseases like Parkinson's, Alzheimer's, and Huntington's represent a significant global health burden.
  • Current treatments for these conditions primarily manage symptoms and are often ineffective due to disease complexity.

Purpose of the Study:

  • To provide an overview of the CRISPR-Cas system's application in neurodegenerative disease therapeutics.
  • To discuss the principles, modeling capabilities, and therapeutic potential of CRISPR-Cas for neurological disorders.

Main Methods:

  • Review of CRISPR-Cas mechanism and its genome-editing capabilities.
  • Exploration of CRISPR-Cas in neurological disorder modeling and target identification.
  • Analysis of gene-based therapies and clinical trials utilizing CRISPR-Cas.

Main Results:

  • CRISPR-Cas enables precise genome editing, offering targeted treatment options for genetic neurological diseases.
  • The system facilitates the development of novel gene-based therapies and aids in understanding disease mechanisms.

Conclusions:

  • The CRISPR-Cas system presents a revolutionary tool for addressing the genetic basis of neurodegenerative diseases.
  • Further research and ethical considerations are crucial for the clinical implementation of CRISPR-Cas in neurological therapeutics.