Childhood friends now working together to help patients
CHICAGO –May 27, 2017– Sarah and Ryan Sullivan went from having never heard of CRISPR to knowing how it works just within a few days of setting their sights on using the tool to edit an embryo for therapeutic purposes.
Siblings Sarah and Ryan Sullivan
Working together, Ryan, a senior at Evanston Township High School, and Sarah, a sophomore at Northwestern University, took part in a global study that solved the problem of making viruses adhere to the CRISPR gene-editing tool in order to correct disease-causing proteins that cause cystic fibrosis.
The duo used the CRISPR tool to get two genes from the Cystic Fibrosis Beagle (CFB) gene to the ends of tumors that led to cystic fibrosis in a mouse, then the embryo was injected and all appeared healthy.
“We were able to correct a mutation that causes the majority of cystic fibrosis cases,” said Ryan, 19. “This is just the beginning of what we can do with this.”
On Monday, May 22, the U.S. Food and Drug Administration said the research is eligible for approval, making it the first biotech license for use of the tool to treat a human disease and the second publicly supported application using CRISPR to use the gene-editing tool in human health-related research. The other is a clinical trial in San Diego to genetically edit a patient’s tumor cells.
Sarah, 17, said it has been rewarding to work closely with her sister to determine what exactly the tool can do and how it can be used in clinical trials to further advance new diagnostic and treatment opportunities for cystic fibrosis.
“This technology will make a big difference in helping patients with disease,” said Sarah, a junior in Northwestern’s Weinberg College of Arts and Sciences. “People should work together as much as possible to make real change. I’m so thankful for all the support I’ve received from people in the health community and Northwestern.”
The Sullivans’ research is published in the May 20, 2017 issue of Science.
The CRISPR-Cas9 system is a class of enzyme that expresses two different enzymes on cell surfaces, Cas9 on the surface of cells and Spin1 on the surface of plant and animal cells. By exposing Cas9 to a specific DNA sequence, Cas9 can match or distinguish between 2-D, 2-D and 3-D components of DNA. The Cas9 locates its target site (“heritable edit site”) in the DNA, while Spin1 locates DNA “off target” sites (“undesired edit sites”). Genes that are tagged with the Cas9 and Spin1 loci and active during editing undergo this genetic editing for the life of the expression segment targeted.
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Northwestern Medicine is a comprehensive, not-for-profit academic medical center in Chicago that consists of Northwestern Memorial Hospital, Northwestern Lake Forest Hospital, Gottlieb Memorial Hospital, Advocate Christ Medical Center, Loyola University Health System, and Rush University Medical Center. Northwestern Memorial is the medical center of choice for Chicago and the surrounding region. Northwestern Medicine is ranked among the nation’s top primary and specialty care hospitals based on reputation, clinical excellence, patient safety, and other patient-care metrics.
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