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ALS - Zarnescu

Daniela Zarnescu (ALS): A315T is a mutant form of the TDP43 protein, identified in people with ALS. Zarnescu's team has engineered a fruit fly model, containing both fly and human proteins, with the A315T mutation. Here, the eyes of adult fruit flies demonstrate the effects of (center) normal and (right) toxic TDP43.
A315T is a mutant form of the TDP43 protein, identified in people with ALS. Zarnescu's team has engineered a fruit fly model, containing both fly and human proteins, with the A315T mutation. Here, the eyes of adult fruit flies demonstrate the effects of (center) normal and (right) toxic TDP43.
Amyotrophic Lateral Sclerosis (ALS)

MDA awarded $375,000 to Daniela Zarnescu, assistant professor in neuroscience at the University of Arizona in Tucson, Ariz., to conduct gene and drug discovery research in a drosophila fruit fly model that carries a mutation in the TDP43 gene associated with a genetic form of human ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

Alterations in TDP43 gene activity in the fruit fly model lead to symptoms that mimic those found in humans with the disease, including the death of nerve cells called motor neurons and the formation of abnormal clumps of cellular material, called inclusions, that contain TDP43 proteins.

Zarnescu's group will conduct genetic screening tests on the flies in order to identify other genes that interact with TDP43, some of which may be involved in disease causation or progression. The group also will screen drugs in search of one or more that can rescue the defects in the fruit fly model.

The work could lead to novel therapeutic targets and approaches for ALS.

"Recognizing the need to fund such projects is not only an important step to ensure important discoveries in the field, but it also shows that MDA is at the forefront of the effort towards identifying new therapies and a potential cure for ALS," Zarnescu said.

For more information read MDA's press release.

Funding for this MDA grant began August 1, 2010.

‹ ALS - Sockanathan up CCD - Muntoni ›

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