Genomic Research in Neurological Disorders

Nearly one out of every seven households in the United States is affected by a neurological disease or disorder. Yet these devastating conditions, which cause the progressive loss of memory, mobility or both, have no cure. Some are impossible to even predict. Genomic technologies can help untangle disorders like Alzheimer, Huntington and Parkinson diseases; ALS; and dementia.

Research: searching for causes of devastating and debilitating neurological disease

The HudsonAlpha Foundation has established the Memory and Mobility (M&M) Program to support genomics research of neurological diseases. Our research includes genome sequencing to pinpoint the genetic causes of Alzheimer disease and dementia. Understanding the genetic causes of Alzheimer disease will enable researchers to better identify promising treatments, and hopefully, a cure. Because neurological diseases are interconnected, discoveries HudsonAlpha researchers make about Alzheimer disease and dementia will also provide clues about other neurological diseases such as ALS, Parkinson disease and Huntington disease.

Discovery: HudsonAlpha identifies eleven new ALS genes

Researchers at HudsonAlpha, in collaboration with other academic institutions and industry scientists, have identified eleven new genes associated with ALS. The genes are now candidates for new drug therapies for the neurodegenerative disease. In addition, HudsonAlpha scientists are applying new analysis techniques to the data from the study in an effort to glean more information and make more crucial discoveries. We are also continuing our study of ALS by sequencing the entire genome of ALS patients (rather than just the genes that make proteins).

Predicting risk: how the environment and genetics affect Parkinson disease

With our colleagues at other institutes, we are studying how and why Parkinson disease develops in order to understand how to stop its progression. Our investigators are examining how environment and genetics combine to affect this debilitating neurodegenerative disease that affects more than one million Americans.

Finding therapies: novel ideas for treating Huntington disease

Huntington disease is caused by an inherited defect in a single gene, so individuals who have this incurable disease carry both the gene with the defect and the gene without the defect – one from each parent. In an effort to develop a therapy to treat this neurodegenerative disease at its genetic source, investigators at HudsonAlpha are using their expertise in understanding how genes are turned on and off. This on/off process, called gene expression, is how an inherited gene becomes a physical trait, whether the trait is a particular feature in a cell or eye color. Our scientists are conducting the most detailed study ever of the Huntington disease gene to examine the possibility of turning off the defective gene, allowing the other gene to function normally. If the Huntington gene can be turned off and the normal gene can continue to function, new therapies to treat the disease could be developed.

To learn more about genomic research in neurological disorders click here to view the 2019-2020 Research Report.