Research & Patient Center

Lab of Dr. Teepu Siddique

The Les Turner ALS Research Laboratory led by Teepu Siddique, MD focuses on the clinical and molecular characteristics of ALS and the development of novel therapeutics. The lab was first dedicated by the Les Turner ALS Foundation in 1979 and has been directed by Dr. Siddique since 1991.

Dr. Siddique, the Les Turner ALS Foundation/Herbert C. Wenske Foundation Professor, is Professor of both Neurology and Cell & Molecular Biology. He is a physician-investigator with expertise in the neurology, molecular genetics, biochemistry and cell biology of neurodegenerative disease. He directs the Division of Neuromuscular Medicine at Northwestern Feinberg School of Medicine and oversees the operations of clinics in the Division including the Lois Insolia ALS Clinic and the MDA clinic. His research in neurogenetics and disease mechanisms of ALS has been generously funded by the National Institutes of Health since 1985. During that time he has fostered the seamless integration of the clinic with the laboratory.

Dr. Siddique pioneered the molecular genetic approach to ALS in 1984, leading a world-wide collaboration which successfully identified the first genetic cause of ALS, the gene SOD1, in 1993. Since then he has employed a range of strategies to identify several genes that cause inherited ALS, particularly ALSIN, FUS, UBQLN2 and SQSTM1, as well as additional genetic loci. His research team developed the first animal model of ALS, the SOD1 transgenic ALS mouse; the first model of upper motor neuron predominant ALS/PLS, the ALSIN knock out mouse; and the first model of ALS/FTD – the UBQLN2 transgenic mouse. Since then the team has developed several other models, using them to study disease mechanism and test potential therapies. His recent discovery of genetic linkage of UBQLN2 and SQSTMI to ALS has shown that those proteins are involved in disease pathology in all types of ALS, sporadic as well as inherited and the ALS-related dementias. This has paved the way for targeted, and therefore effective, treatments of ALS. Most recently, the Siddique team identified a new gene for familial Parkinson disease (Nature Genetics, 2016).

The journal Neurology, the most widely read and quoted journal in the field, cited both Dr. Siddique’s identification of the first causative gene in ALS in 1993 and his team’s 2010 report of malfunction in the ubiquitin-proteosome system as the two real paradigm shifts in understanding ALS.

The Siddique Laboratory maintains a Neurologic Diseases Registry that contains more than 16,000 blood samples and 120 autopsied ALS cases.

Dr. Teepu Siddique

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Recent Publications

Neurology Genetics
Compound heterozygote mutations in SPG7 in a family with adult-onset primary lateral sclerosis

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