In vivo CRISPR Screening for Modifiers of Mutant Huntingtin Levels

Women’s Council Recipient
Myriam Heiman, Ph.D.
Massachusetts Institute of Technology

Huntington’s disease (HD) is an incurable brain disease that afflicts roughly 1 in every 10,000 people in the United States. HD is associated with movement problems, cognitive deficits, and psychiatric symptoms. HD most often strikes in middle age and leads to death in 10-20 years after symptom onset. Only symptomatic therapies exist to treat HD, and they are targeted to movement abnormalities, and do not help much with cognitive deficits or psychiatric symptoms. None of the existing therapies can stop of the progression of HD. Thus there is a clear need for new therapeutics that can stop the halt of HD, and even to reverse its course. Recent brain research studies have offered some hope. These studies have shown that lowering the levels of the mutant HD DNA product can halt and even reverse the progression of HD. Armed with this knowledge, we hope to identify other genes in DNA that can lead to lowered levels of the HD DNA product. To do so, we will conduct a genetic screen – systematically asking, gene by gene across all of the DNA, which genes can lower the mutant HD DNA product. Once identified, genes that we show lead to lower HD DNA product levels will be promising new therapeutic  targets.

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