JAN 03, 2017 06:39 AM PST
Synthetic DNA Technology Can Repair Genetic Defects
WRITTEN BY: Xuan Pham
4 9 297

While CRISPR gene editing has dominated the genome craze for the last few years, there's a new next-generation platform that's been in the works and is showing big promise for the treatment of a blood disorder.

The system, developed by researchers at Carnegie Mellon University and Yale University, relies on synthetic nucleotide molecules called peptide nucleic acid (PNA). Unlike CRISPR, which uses molecular scissors to ‘cut-and-paste' the desired genetic changes, the PNA system is designed to open up double-stranded DNA. Then, other components of the system coax the host's machinery to repair the faulty genetic change.

In the lab, researchers were able to successfully target a defect in that causes beta thalassemia in a mouse model. The team noted that the system has high specificity for the desired target site and produced a significant change in hemoglobin production.

Watch the video to learn more!
About the Author
  • I am a human geneticist, passionate about telling stories to make science more engaging and approachable. Find more of my writing at the Hopkins BioMedical Odyssey blog and at TheGeneTwist.com.
You May Also Like
MAY 08, 2018
Videos
MAY 08, 2018
Virtual Reality Theme Park Opens in China
China's first virtual reality theme park, called Oriental Science Fiction Valley, opened at the end of April 2018. It has a sci-fi focus and offers guests
MAY 10, 2018
Videos
MAY 10, 2018
Is It Candy or Vape Juice?
Vaping is extremely popular with young people, sometimes even middle schoolers, but the FDA has partnered with the FTC to warn vape manufacturers that thei
MAY 14, 2018
Videos
MAY 14, 2018
Running on a Treadmill vs. Running Outdoors
Is it better to run on a treadmill or outdoors? Apparently, the differences are not only personal preferences but health advantages as well. For one, runni
MAY 16, 2018
Videos
MAY 16, 2018
There Could Be Life on Venus After All...
Of all the places in the solar system where you might find alien life, Venus seems unlikely. But counting Venus out merely because of its hellish condition
MAY 17, 2018
Videos
MAY 17, 2018
How Musical Instruments Boost Brain Function
Music and the brain are often part of the same scientific research projects. How music is processed in the brain and how we learn to recognize and replicat
MAY 22, 2018
Videos
MAY 22, 2018
FDA Warns Companies About Generic Drug Supply
With new drugs being developed, many health conditions and illnesses now have better treatments. New drugs are expensive, however, due to the all of the mo
Loading Comments...