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Related Concept Videos

Genome-wide Association Studies-GWAS01:11

Genome-wide Association Studies-GWAS

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Genome-wide association studies or GWAS are used to identify whether common SNPs are associated with certain diseases. Suppose specific SNPs are more frequently observed in individuals with a particular disease than those without the disease. In that case, those SNPs are said to be associated with the disease. Chi-square analysis is performed to check the probability of the allele likely to be associated with the disease.
GWAS does not require the identification of the target gene involved in...
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Competitive Genomic Screens of Barcoded Yeast Libraries
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The NIH Somatic Cell Genome Editing program.

Krishanu Saha1,2,3,4, Erik J Sontheimer5, P J Brooks6

  • 1Department of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI, USA. ksaha@wisc.edu.

Nature
|April 8, 2021
PubMed
Summary
This summary is machine-generated.

The NIH Somatic Cell Genome Editing (SCGE) Consortium is developing tools and methods for safer genome editing in patients. This initiative aims to accelerate the creation of new therapies for various diseases by validating genome editing technologies.

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Area of Science:

  • Biotechnology
  • Genomics
  • Medical Research

Background:

  • Genome editing holds promise for improving human health.
  • Current methods require further development for clinical application, especially for hard-to-reach tissues.

Purpose of the Study:

  • To accelerate the development of safer and more effective somatic cell genome editing (SCGE) methods.
  • To establish rigorous benchmarking and validation approaches for genome editing technologies.
  • To create a comprehensive toolkit for the biomedical research community.

Main Methods:

  • Developing and validating genome editing tools for somatic cells.
  • Measuring genome modifications and their functional consequences in human cells.
  • Conducting third-party validation in animal models (small and large).
  • Developing in vivo cell tracking methods.

Main Results:

  • The consortium plans to assemble validated datasets, genome editors, delivery technologies, and animal models into an SCGE Toolkit.
  • The toolkit will include methods for inducing and measuring genome modifications and assessing downstream effects.
  • Validation will involve rigorous testing in preclinical models.

Conclusions:

  • The SCGE Toolkit will be widely disseminated to accelerate clinical development.
  • This initiative aims to advance therapeutic applications of genome editing for diverse conditions.
  • Standardized validation and accessible tools are crucial for translating genome editing into clinical practice.