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Related Concept Videos

CRISPR01:59

CRISPR

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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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CRISPR/Cas9 Genome Editing01:28

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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...
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Homologous Recombination02:31

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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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CRISPR and crRNAs02:53

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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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Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells
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CRISPR-Cas9 for treating hereditary diseases.

Indra Mani1

  • 1Department of Microbiology, Gargi College, University of Delhi, New Delhi, India.

Progress in Molecular Biology and Translational Science
|June 15, 2021
PubMed
Summary

Genome editing, particularly CRISPR-Cas9 technology, offers a promising approach for treating genetic diseases by correcting DNA mutations. This tool is being investigated for various inherited disorders, showing potential for improved human health.

Keywords:
CRISPRCas-9Genetic diseasesGenome editingMutations

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Area of Science:

  • Genetics and Molecular Biology
  • Biotechnology
  • Medical Research

Background:

  • Genome editing tools allow for precise modification of DNA in cells and organisms.
  • Understanding physiological responses to genetic alterations is crucial for disease research.
  • Emerging technologies like CRISPR-Cas9 have revolutionized biological sciences.

Purpose of the Study:

  • To provide general information on the application of genome editing tools for treating genetic diseases.
  • To explore the potential of CRISPR-Cas9 technology in addressing hereditary conditions.
  • To discuss current advancements, challenges, and future prospects of CRISPR-Cas9 in disease treatment.

Main Methods:

  • Utilizing clustered regularly short palindromic repeats (CRISPR) and CRISPR-associated protein 9 (CRISPR-Cas9) for DNA mutation editing.
  • Applying genome editing techniques in both in vitro (cell) and in vivo (animal) models.
  • Investigating the efficacy of CRISPR-Cas9 in a range of human hereditary diseases.

Main Results:

  • CRISPR-Cas9 is being actively used to study and potentially treat numerous genetic diseases.
  • The technology shows promise for conditions including hemophilia, cystic fibrosis, Alzheimer's, and Duchenne muscular dystrophy.
  • Genome editing applications extend to improving human health beyond inherited disorders.

Conclusions:

  • CRISPR-Cas9 technology represents a significant advancement in the treatment of genetic diseases.
  • Ongoing research focuses on overcoming challenges and realizing the full therapeutic potential of genome editing.
  • The future prospects for CRISPR-Cas9 in medicine are substantial, offering hope for novel treatments.