21 Jul Magic of science: FSU researchers use gene editing to tackle facioscapulohumeral muscular dystrophy
Roughly 1 out of 15,000 people born suffer from Facioscapulohumeral Muscular Dystrophy (FSHD), a rare disease that starts with the progressive weakening of facial muscles and then the shoulders and upper body. By the time patients reach their 50s, around one-fifth of them are wheelchair bound.
Now, a team of Florida State University researchers has found that using sophisticated gene-editing techniques, they can essentially turn off or remove part of the genetic code that leads to the disease — a move that opens the door for potential therapies to reverse or stop progression of the disease.
The work is published in Scientific Reports.
“Applying genome engineering to excise the pathogenic element in FSHD was enormously challenging due to the repetitive nature of the chromosomal region responsible for the disease, but I was delighted to be able to obtain the result we were hoping for,” said former FSU graduate student Sunny Das, the lead author of the study. “My hope is that these results will open the door to the very real potential of a new therapeutic possibility.”