The following is an excerpt from Dana G. Smith, PhD | September 1, 2016 | gladstone.org |
The pace of science is accelerating. In the past, research methods remained the same for decades. Now, a new technology emerges every few years that disrupts the status quo of biological science. The most recent breakthrough is CRISPR—a tool to edit the genome of a human cell more precisely and efficiently than ever before.
CRISPR has enormous potential to cure intractable diseases. At the Gladstone Institutes, scientists are using the technology to advance scientific knowledge and pursue new therapies for heart disease, HIV, dementia, blindness, and more. With CRISPR, they have improved research methods and developed better models of genetic disorders. They are also embarking on a precision medicine approach to treat these debilitating conditions.
“CRISPR is a real game-changer. It is a huge jump forward from previous genome editing techniques,” said Gladstone Senior Investigator Bruce Conklin, MD, an early adopter of the technology. “With CRISPR, we can accomplish research that we never before thought possible.”
What is CRISPR?
At its most basic, CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a string of RNA. When introduced into a cell, the RNA seeks an identical series of letters in the DNA of the cell. Scientists can design the precise letter sequence they want to target, and use CRISPR to home in on that specific part of the genome. The technique enables researchers to pinpoint a single series of letters out of the three billion that make up human DNA.
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