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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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What is Genetic Engineering?00:49

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CRISPR and crRNAs02:53

CRISPR and crRNAs

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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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Homologous Recombination02:31

Homologous Recombination

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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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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.
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Genome Editing in Mammalian Cell Lines using CRISPR-Cas
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Genome Editing in Mammalian Cell Lines using CRISPR-Cas

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Therapeutic genome engineering via CRISPR-Cas systems.

Ana M Moreno1, Prashant Mali1

  • 1Department of Bioengineering, University of California San Diego, San Diego, CA, USA.

Wiley Interdisciplinary Reviews. Systems Biology and Medicine
|February 16, 2017
PubMed
Summary

Genome and epigenome engineering using clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated (Cas) systems offer new therapeutic strategies for human diseases. This review explores CRISPR-Cas applications, challenges, and future directions in genome engineering.

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Area of Science:

  • Genomic Medicine
  • Molecular Biology
  • Biotechnology

Background:

  • Genetic variations drive organismal diversity and disease.
  • Advancements in understanding disease genetics necessitate novel therapeutic platforms.
  • Genome and epigenome engineering hold potential for treating diseases and enhancing resistance.

Purpose of the Study:

  • To review therapeutic genome engineering applications.
  • To focus specifically on CRISPR-Cas toolsets.
  • To summarize current work and outline future directions.

Main Methods:

  • Review of existing literature on CRISPR-Cas systems.
  • Analysis of therapeutic genome engineering strategies.
  • Identification of challenges and future prospects in the field.

Main Results:

  • CRISPR-Cas systems have revolutionized nucleic acid targeting.
  • These systems provide powerful tools for genome and epigenome engineering.
  • Significant progress has been made in therapeutic applications.

Conclusions:

  • CRISPR-Cas technology is transforming the landscape of human disease treatment.
  • Further development is needed to address key challenges.
  • Future directions include refining CRISPR-Cas for enhanced therapeutic efficacy and safety.