ATTENTION: Annual institutional progress report forms will be sent to all participating Institutions on Mon, Feb 6. These forms will need to be completed by all PI’s SC INBRE faculty and Pilot Project recipients and returned to John Clarkson. These are extremely important as they are used to complete the annual RPPR for NIH. Please return completed forms no later than Mon, March 6. If you have any questions, please contact John (email) or Dr. Pirisi-Creek (email).

For gene therapy research, the perennial challenge has been devising a reliable way to insert safely a working copy of a gene into relevant cells that can take over for a faulty one. But with the recent discovery of powerful gene editing tools, the landscape of opportunity is starting to change. Instead of threading the needle through the cell membrane with a bulky gene, researchers are starting to design ways to apply these tools in the nucleus—to edit out the disease-causing error in a gene and allow it to work correctly.

CRISPR/Cas9 uses small “guide RNA” molecules together with a scissor-like enzyme to find and snip the specific, incorrect DNA sequence in just the right spot. Once the DNA is cut, the cell completes the edit using the correct gene sequence from a DNA fragment supplied by the researchers as its template. Think of it as “find and replace” for the genome.

While CRISPR approaches come with tremendous potential advantages in precision, other gene therapy approaches continue to show promise too. In fact, a clinical trial under way at NIH and other sites around the country is now testing a more-traditional approach to chronic granulomatous disease (CGD) gene therapy. It uses an inactivated, non-infectious virus to insert a working gene into the cells of CGD patients. It’s too soon to know how well it will ultimately work, but early indications are encouraging.

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What do you think of gene therapy to treat diseases?

• It's the future of medicine.
• I would like to learn more.
• It is not as good as other, more traditional medicine and treatments.
• I don't have an opinion on the subject.