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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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Gene Therapy00:59

Gene Therapy

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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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.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
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What is Genetic Engineering?00:49

What is Genetic Engineering?

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Overview
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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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Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
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CRISPR/Cas gene therapy.

Baohong Zhang1

  • 1Department of Biology, East Carolina University, Greenville, North Carolina, USA.

Journal of Cellular Physiology
|September 22, 2020
PubMed
Summary
This summary is machine-generated.

Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated enzyme (Cas) gene editing shows promise for genetic diseases. Research focuses on improving CRISPR/Cas9 specificity and delivery to overcome challenges like off-target effects for clinical use.

Keywords:
CRISPR/Cas9animal modelgene therapygenetic diseasegenetic disordergenome editing

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • CRISPR/Cas is a prokaryotic adaptive immune system repurposed for genome editing.
  • CRISPR/Cas9 is a powerful tool for treating genetic diseases like cardiovascular disorders, neurological conditions, and cancers.
  • This technology is also valuable for creating animal models of human genetic disorders, especially those with point mutations.

Purpose of the Study:

  • To review the current applications and challenges of CRISPR/Cas9 genome editing in human disease treatment and genetic modeling.
  • To highlight the need for further research into improving the specificity and delivery of CRISPR/Cas9 systems.
  • To address concerns regarding toxicity, immune response, and off-target effects.

Main Methods:

  • Review of existing literature on CRISPR/Cas9 applications in preclinical and clinical studies.
  • Analysis of strategies for enhancing CRISPR/Cas9 specificity, including gRNA design and Cas enzyme selection.
  • Evaluation of advancements in CRISPR/Cas9 delivery systems for targeted tissue/cell modification.

Main Results:

  • CRISPR/Cas9 has demonstrated significant potential in treating various genetic diseases and creating disease models.
  • Key challenges include potential toxicity, immune responses, efficient delivery, and off-target mutations.
  • Designing highly specific guide RNAs (gRNAs) and utilizing high-specificity Cas enzymes are crucial for minimizing off-target effects.

Conclusions:

  • CRISPR/Cas9 technology holds immense promise for gene therapy and disease modeling.
  • Addressing challenges such as off-target effects through improved specificity and delivery methods is essential for clinical translation.
  • Continued research and development are necessary to fully realize the therapeutic potential of CRISPR/Cas9.