JUL 10, 2018 5:18 PM PDT

New Tools in the CRISPR Kit

WRITTEN BY: Carmen Leitch

Some genetic diseases are more straightforward than others. In cases like cystic fibrosis or Marfan syndrome, a mutation makes a gene defective, and that causes symptoms in a patient that are characterized as a disease. But other kinds of genetic diseases can involve a complex interplay of genes and sometimes, environmental factors, that all work together to create symptoms in a patient. Heart disease and type 2 diabetes are examples of complex diseases.

When using a tool like CRISPR, making a single gene edit can be all that’s necessary to produce or rescue a disease phenotype, but for studying complex diseases a more refined tool is needed. Scientists at UCSF created altered versions of CRISPR so that genes can be manipulated in other ways; CRISPRi can lower gene expression levels, and CRISPRa can activate genes, as explained in the video.

About the Author
  • Experienced research scientist and technical expert with authorships on over 30 peer-reviewed publications, traveler to over 70 countries, published photographer and internationally-exhibited painter, volunteer trained in disaster-response, CPR and DV counseling.
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