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What is Amyotrophic lateral sclerosis (ALS)?

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder that affects brain and spinal cord nerve cells responsible for controlling muscle movement. The majority of ALS cases are sporadic (no family history of the disease), but 5-10% of cases are familial (caused by an inherited genetic mutation). Current treatments help control symptoms and may slow progression of the disease, but a cure for this devastating disease is greatly needed.

ALS Therapeutic Strategies

Synthetic MicroRNAs May Safely Treat Patients With ALS

Mutations in the superoxide dismutase 1 (SOD1) gene account for 20% of familial ALS cases. The labs of Drs. Mueller and Brown have demonstrated that a gene therapy strategy—using a synthetic microRNA targeting the SOD1 gene—can silence the SOD1 protein in motor neurons in animal models without causing any adverse effects. Their findings indicate the ALS treatment should be safe in humans.

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Amyotrophic Lateral Sclerosis (ALS) Researchers

Robert Brown, MD, DPhil

Robert H. Brown Jr., DPhil, MD

The Leo P. and Theresa M. LaChance Chair in Medical Research and Chair of the Department of Neurology

Brown Lab

John E. Landers, Ph.D.

Professor of Neurology

Landers Lab

chris mueller phd

Chris Mueller, PhD

Associate Professor

Mueller Lab

Daryl Bosco, PhD

Associate Professor

Bosco Lab

Fen-Biao Gao, PhD

Professor, Governor Paul Cellucci Chair in Neuroscience Research

Fen-Biao Gao Lab

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

What is Amyotrophic lateral sclerosis (ALS)?