Our Plan to Help:
Sr. Hamel will compare how the extent of toxic RNA accumulation and protein dysfunction in the muscle cell nucleus relates to signs and symptoms of patients.
The Potential Impact of this Research:
Current therapies under investigation for the treatment of myotonic dystrophy are designed to decrease the levels of toxic RNA and thereby release trapped proteins. A better understanding of how and to what extent the RNA is toxic for the cell will help to define treatment goals for further drug development. This knowledge about RNA toxicity may also be of use for other genetic disorders caused by toxic RNAs.
How You Can Help:
By donating to Dr. Hamel's research you can assist with the development of drug treatments for patients with myotonic dystrophy.