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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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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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Crystal Growth: Principles of Crystallization01:25

Crystal Growth: Principles of Crystallization

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Crystallization is a phase transformation process in which crystals are precipitated from a supersaturated solution or formed from other sources. During crystallization, atoms or molecules arrange themselves into a well-defined, rigid crystal lattice to minimize energy.
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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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Patch Clamp01:18

Patch Clamp

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Many fundamental cell functions such as muscle contraction and nerve transmission rely on the electrical signals produced by the movement of positively and negatively charged ions across the cell membrane. One competent method to record current flowing across the whole cell or single ion channel is the patch-clamp technique.
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Issues And Trends In Healthcare Delivery System01:29

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The issues and trends in healthcare delivery are constantly changing. The COVID-19 pandemic is one recent issue that wreaked havoc on healthcare systems, causing a shortage of healthcare workers, high demand for medicines and supplies, and increased medical expenditure due to a lack of insurance. Other issues include rising healthcare costs and care fragmentation.
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Genome-Wide CRISPR Screen for Unveiling Radiosensitive and Radioresistant Genes
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Genome-Wide CRISPR Screen for Unveiling Radiosensitive and Radioresistant Genes

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CRISPRing future medicines.

Laure Grand Moursel1, Mijke Visser1, Geraldine Servant1

  • 1Charles River Laboratories, Leiden, The Netherlands.

Expert Opinion on Drug Discovery
|December 16, 2020
PubMed
Summary
This summary is machine-generated.

CRISPR technology offers a fast and affordable method for mammalian genome engineering, revolutionizing biomedical research. This tool accelerates drug discovery and enhances cell and gene therapies by simplifying genome modification.

Keywords:
CRISPRCRISPR-engineered animal and cellular modelscell and gene therapydrug discoveryfunctional genomic screeninggenome engineeringtarget discoverytarget validation

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Area of Science:

  • Biomedical Research
  • Genomic Engineering

Background:

  • CRISPR technology enables rapid and cost-effective mammalian genome engineering.
  • Its adoption in biomedical research is accelerating.
  • It holds significant potential for drug discovery and cell/gene therapies.

Purpose of the Study:

  • To review the transformative impact of CRISPR technology on drug discovery.
  • To highlight its applications from target identification to validation.
  • To discuss its role in advancing cell and gene therapy development.

Main Methods:

  • Review of CRISPR technology applications in drug discovery.
  • Analysis of its use in in vitro and in vivo models.
  • Examination of its role in cell and gene therapy.

Main Results:

  • CRISPR technology streamlines genome engineering for early-stage drug development.
  • It positively impacts target identification and validation processes.
  • It is a key enabling technology for cell and gene therapies.

Conclusions:

  • CRISPR is becoming the standard for genome engineering in drug discovery.
  • It is indispensable for modern drug discovery pipelines.
  • Further refinement will solidify its role in drug discovery and cell/gene therapy.