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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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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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Retroviruses have a single-stranded RNA genome that undergoes a special form of replication. Once the retrovirus has entered the host cell, an enzyme called reverse transcriptase synthesizes double-stranded DNA from the retroviral RNA genome. This DNA copy of the genome is then integrated into the host’s genome inside the nucleus via an enzyme called integrase. Consequently, the retroviral genome is transcribed into RNA whenever the host’s genome is transcribed, allowing the...
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The Antiviral System of Bacteria and Archaea: CRISPR01:23

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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...
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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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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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CRISPR therapy towards an HIV cure.

Elena Herrera-Carrillo1, Zongliang Gao1, Ben Berkhout1,2

  • 1Department of Medical Microbiology Laboratory of Experimental Virology Amsterdam UMC, AMC, University of Amsterdam, Amsterdam, the Netherlands.

Briefings in Functional Genomics
|November 12, 2019
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Summary

RNA interference (RNAi) and CRISPR gene editing offer promising therapeutic strategies for HIV. Optimizing their design is key to overcoming limitations like efficiency, specificity, and delivery for effective treatment.

Keywords:
CRISPR-CasHIVRNA interferencegene therapylentiviral vectorpolymerase III promoter

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Area of Science:

  • Molecular Biology
  • Gene Therapy
  • Virology

Background:

  • RNA interference (RNAi) and clustered regularly short palindromic repeats (CRISPR) are powerful tools for modifying gene expression.
  • These technologies show potential as therapeutic agents for infectious diseases, including HIV.
  • Both platforms share similarities and face challenges in clinical applications.

Purpose of the Study:

  • To review the advantages and limitations of RNAi and CRISPR-Cas9 as anti-HIV strategies.
  • To focus on key aspects such as efficiency, specificity, off-target effects, and delivery methods.
  • To discuss how optimal design can overcome current therapeutic challenges.

Main Methods:

  • Comparative analysis of RNAi and CRISPR-Cas9 platforms.
  • Review of existing literature on anti-HIV strategies utilizing these technologies.
  • Focus on evaluating efficiency, specificity, safety, and delivery.

Main Results:

  • Both RNAi and CRISPR-Cas9 present distinct advantages and limitations for HIV therapy.
  • Efficiency, specificity, and off-target effects are critical parameters for therapeutic success.
  • Delivery methods remain a significant hurdle for effective in vivo application.

Conclusions:

  • Further research and optimized design of RNAi and CRISPR-based therapies are necessary for combating HIV.
  • Addressing limitations in specificity and delivery will enhance the therapeutic potential of these gene-editing tools.
  • These technologies hold promise for future infectious disease treatments if challenges are effectively managed.