A path to treat currently untreatable cases of cystic fibrosis is clearly achievable, study suggests
An experimental drug reported in
Nature Communications suggests that a path is clearly achievable to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent of cystic fibrosis patients, as well as patients with other genetic diseases, including Duchenne muscular dystrophy, β-thalassemia and numerous types of cancers, that are also caused by nonsense mutations.
The drug is a small molecule with a novel mechanism of action, say David Bedwell, Ph.D., and Steven Rowe, M.D., MSPH, co-senior authors. Bedwell is professor and chair of the University of Alabama at Birmingham Department of Biochemistry and Molecular Genetics, and Rowe is a professor in the UAB Department of Medicine.
A small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations
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Small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations
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By Robin DeMonia
A Southern Research neuroscience lab has received new federal grants totaling almost $3.9 million to advance its study of Parkinson’s disease and other neurological disorders.
The grants from the National Institute of Neurological Disorders and Stroke include $3.3 million to study Parkinson’s and $594,000 for research related to frontotemporal dementia, which is similar to Lou Gehrig’s disease – amyotrophic lateral sclerosis (ALS).
Both grants will support a lab led by Rita Cowell, fellow and chair of the Department of Neuroscience in Southern Research’s Drug Discovery Division.
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