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Therapeutic Strategies

 

Gene Therapy to Silence HTT


The labs of Drs. Mueller and Aronin are partnering to develop adeno-associated virus (AAV) vector-based gene therapy approaches using a synthetic microRNA targeting HTT to silence (or turn off) the disease-causing gene.

 

huntington's disease

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Huntington’s Disease Investigators

Neil Aronin MD

Neil Aronin MD

Professor of medicine, co-director of the Neurotherapeutics Institute

Aronin Lab

John E. Landers, Ph.D.

John E. Landers, Ph.D.

Professor of Neurology

Landers Lab

Miguel Sena-Esteves, Ph.D.

Associate Professor, Department of Neurology

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Clinical Trials

At the Li Weibo Institute for Rare Diseases Research, our researchers and clinicians are dedicated to finding new treatment options for patients. Our clinical trials program is another way we continue to advance treatments and make new discoveries. If you or someone you know may be interested in a clinical trial, please subscribe for updates and you will be notified when we open up a new trial.

 

Research That Gives Hope

Understanding Huntington’s disease

By Neil Aronin, MD

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Huntington’s disease is interesting because it is a genetic disease caused by one allele of one gene. We know which gene it is and what the mutation is (an expansion of the CAG repeat region in Exon 1 of the Huntingtin gene). By comparison, Parkinson’s disease and Alzheimer’s disease could be caused by one gene or many genes, and environmental factors...

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