Advertisement
Advertisement
dystrophin
/ ˈdɪstrəfɪn /
noun
- a protein, the absence of which is believed to cause muscular dystrophy
Example Sentences
The effects were that muscular dystrophin was alleviated throughout the body.
Food and Drug Administration gave the treatment— in which viruses deliver a gene for a short form of the muscle protein dystrophin into muscle tissue—a tentative “accelerated approval” limited to 4- and 5-year-olds possessing a dystrophin gene mutation.
But because the gene for the missing dystrophin protein is so large, a smaller version of the gene is used.
Because of a mutation in the gene for dystrophin, DMD patients lack functioning copies of the huge protein that serves as a shock absorber inside muscle fiber cells.
The DNA encoding dystrophin is too large to package into the AAVs widely used in gene therapy.
Advertisement
Advertisement
Advertisement
Advertisement
Browse