The Miller Laboratory is dedicated to understanding neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and dementias in order to develop new, effective, and safe treatments. We focus on translational neuroscience, new therapeutic approaches for neurodegenerative diseases, and precision medicine.


ALS Research

The Miller Lab seeks to understand the pathogenesis of ALS with basic studies focused on SOD1, C9ORF72, TBK-1,  miRNA, protein misfolding, protein kinetics, and genetic modifiers of disease. We employ RNA-targeted therapeutic strategies, mainly using antisense oligonucleotides.

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Dementia Research

The Miller Lab studies how the various isoforms of the protein tau affect neurodegeneration. We employ RNA-targeted therapeutic strategies using antisense oligonucleotides to develop therapies for dementias involving the tau protein as well as for TREM-2.

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Promising Results for Investigational Treatments for Inherited ALS

Two preliminary studies were published simultaneously in the New England Journal of Medicine, both aimed at reducing superoxide dismutase 1 (SOD1) protein levels in people who had amyotrophic lateral sclerosis (ALS) due to SOD1 mutations. In the first study, a phase I/II trial of intrathecal tofersen, Timothy Miller, MD, PhD, of Washington University in St. […]


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