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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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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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Microorganisms in Medicine and Therapeutics01:29

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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What is Genetic Engineering?00:49

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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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A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
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CRISPR-based therapeutics: current challenges and future applications.

Ashley E Modell1, Donghyun Lim1, Tuan M Nguyen1

  • 1Chemical Biology and Therapeutics Science, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Department of Medicine, Harvard Medical School, Boston, MA 02115, USA; Divisions of Renal Medicine and Engineering, Brigham and Women's Hospital, Boston, MA 02115, USA.

Trends in Pharmacological Sciences
|December 25, 2021
PubMed
Summary
This summary is machine-generated.

CRISPR gene editing is revolutionizing therapeutics by aiding drug discovery and developing novel treatments. Researchers are overcoming in vivo limitations of CRISPR-Cas9 to advance new therapies.

Keywords:
CRISPR-Cas9base editingcell-based therapeuticsgenome engineeringprime editing

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Area of Science:

  • Biotechnology
  • Genomics
  • Molecular Biology

Background:

  • The discovery of CRISPR-associated (CRISPR-Cas) nucleases has transformed therapeutic development.
  • CRISPR technology offers potential for drug discovery, mechanism of action identification, and ex vivo therapeutics.

Purpose of the Study:

  • To discuss the advancements and challenges of CRISPR-Cas systems in both conventional and emerging therapeutic applications.
  • To highlight strategies for overcoming limitations of CRISPR-Cas9 for in vivo therapeutics.

Main Methods:

  • Review of CRISPR-based screening for drug discovery and identification of escape mutants.
  • Discussion of CRISPR-Cas advancements in ex vivo therapeutics, such as cell replacement therapies.
  • Exploration of engineered CRISPR-Cas9 systems, including inhibitors, degradable controls, biomolecule conjugates, and base editors.

Main Results:

  • CRISPR-Cas systems are significantly impacting drug discovery and the development of novel therapeutic strategies.
  • CRISPR-based screens efficiently identify drug mechanisms and resistance pathways.
  • Engineered CRISPR-Cas9 variants and delivery methods are addressing in vivo therapeutic challenges.

Conclusions:

  • CRISPR-Cas technology holds immense promise for revolutionizing medicine, from drug discovery to in vivo and ex vivo treatments.
  • Overcoming limitations such as delivery, immunogenicity, and off-target effects is crucial for realizing the full therapeutic potential of CRISPR-Cas9.
  • Continued innovation in CRISPR-Cas systems will drive the next generation of advanced therapeutics.