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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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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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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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The Antiviral System of Bacteria and Archaea: CRISPR01:23

The Antiviral System of Bacteria and Archaea: CRISPR

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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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Methods Of Healthcare Delivery System01:26

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At the different levels of the healthcare system, we see varying methods of healthcare used. These methods include managed care systems, case management, and primary healthcare.
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Eukaryotic cells use different mechanisms to eliminate toxic waste obsolete and worn-out substances. Lysosomes play a pivotal role in this, and hence, these substances are carried to the lysosome from other parts of the cell and extracellular space through different pathways. The most elaborately studied pathways to the lysosome are the endocytic pathways.
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Using CRISPR/Cas9 to Knock Out GM-CSF in CAR-T Cells
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Nanoparticles for CRISPR-Cas9 delivery.

Zachary Glass1, Yamin Li1, Qiaobing Xu2

  • 1Department of Biomedical Engineering, Tufts University, Medford, MA, USA.

Nature Biomedical Engineering
|August 28, 2018
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Summary
This summary is machine-generated.

Gold nanoparticles precisely corrected the Duchenne muscular dystrophy DNA mutation in mice. This gene editing approach showed minimal off-target effects, offering a promising therapeutic strategy.

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Area of Science:

  • Biomedical Engineering
  • Genetics
  • Nanotechnology

Background:

  • Duchenne muscular dystrophy (DMD) is a severe genetic disorder.
  • Current treatments for DMD are limited and primarily focus on symptom management.
  • Gene editing offers a potential curative approach for genetic diseases like DMD.

Purpose of the Study:

  • To investigate the efficacy of gold nanoparticles as a delivery system for CRISPR gene editing components.
  • To correct the specific DNA mutation responsible for Duchenne muscular dystrophy in a mouse model.
  • To evaluate the safety and specificity of this nanoparticle-mediated gene editing approach.

Main Methods:

  • Utilized gold nanoparticles functionalized to carry CRISPR-Cas9 gene editing tools.
  • Administered the gold nanoparticle-CRISPR complex to mice with the Duchenne muscular dystrophy mutation.
  • Assessed gene editing efficiency and analyzed for off-target mutations at the DNA level.

Main Results:

  • Successfully corrected the causative DNA mutation for Duchenne muscular dystrophy in the studied mouse model.
  • Observed minimal to negligible off-target editing events, indicating high specificity.
  • Demonstrated the potential of gold nanoparticles to safely and effectively deliver gene editing machinery.

Conclusions:

  • Gold nanoparticle-mediated CRISPR delivery is a viable strategy for correcting the DMD mutation in vivo.
  • This approach shows promise for developing targeted and safe gene therapies for Duchenne muscular dystrophy.
  • Further research is warranted to translate this finding into human clinical applications.