When Patrick Doherty recently found out that he was diagnosed with transthyretin amyloidosis, the same disease that killed his father, he was devastated, according to NPR. “It’s terrible prognosis…it’s just dreadful,” Doherty said. So, when he heard about a new potential treatment, he quickly volunteered.
The new treatment involves a relatively-new approach to editing our DNA directly, called CRISPR (short for clustered regularly interspaced short palindromic repeats). CRISPR can be used to directly modify and correct the malformed DNA causing illness. It has also previously been used to correct the blood ailments sickle cell disease and beta thalassemia.
The technique used to treat Doherty’s disease is still in its early stages, and the study’s doctors reiterate that much more work needs to be done and more patients need to undergo treatment to further confirm its efficacy. However, Doherty and other participants are pleased to report that the revolutionary CRISPR treatment is showing signs of success. “I definitely feel better,” Doherty said. “I’m speaking to you from upstairs in our house. I climbed stairs to get up here. I would have been feeling breathless. I’m thrilled.”