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. Teepu Siddique was the founder of the Neuromuscular Disorders Program and the  Neurogenetics Laboratory at Northwestern in 1991 with continuous support from the Les Turner ALS Foundation. His group’s work focuses on the clinical and molecular characteristics of ALS and the development of novel therapeutics. Dr. Siddique, the Les Turner ALS Foundation/Herbert C. Wenske Foundation Professor, is Professor of both Neurology and Cell & Molecular Biology at Northwestern University. He is a physician-neuroscientist with expertise in the neurology, molecular genetics, biochemistry, cell biology and animal modeling of human neurodegenerative disease. Since 1991, Dr. Siddique has had a large clinical practice at the Lois Insolia ALS Clinic at the Les Turner Center at Northwestern Medicine with a specialty in familial ALS cases.

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 discoveries of UBQLN2 and SQSTMI as causes of ALS and their signature involvement in the pathology of all types of ALS and ALS-dementia, sporadic as well as inherited, has paved the way for rationally targeted, and therefore effective, treatments of ALS. In 2014 the Siddique lab published a novel class of compounds that reduces levels of SOD1 in cells and tissues, the first tier of drugs targeted for SOD1-ALS.

With current support from the National Institute of Environmental Health Sciences for study of gene-environment interaction in ALS, Dr. Siddique’s laboratory has identified serum markers associated with sporadic ALS.

A common feature of all types of ALS is the disruption of quality control of protein homeostasis, which often results in the accumulation of misfolded protein aggregates. The lab has recently been awarded an “idea” grant by the Department of Defense to examine whether activation of the ubiquitin pathway will enhance clearance of toxic proteins such as mutant SOD1 and slow disease progression in ALS.

Applying the experience gained from their study of molecular genetics of ALS, in 2016 Drs. Han-Xiang Deng and Siddique published a new gene for familial Parkinson disease, another neurodegenerative disorder.

Two preeminent authorities in ALS, Drs. Stanley Appel and Lewis P. Rowland, in an Editorial Comment in Neurology (2012), highlighted the two paradigm shifts in understanding of ALS since 1991 by citing Dr. Siddique’s contributions. They were referring to Dr. Siddique and colleague’s application of molecular genetics and the discovery of the first causative gene in ALS in 1993 and the team’s 2011 reports of the first causative links between the ubiquitin-proteasome system (ubiquilin2) and the autophagosome-lysosome system (SQSTM1).

Dr. Siddique has received national and international recognition, including the Rattigan Medal from the University of the Punjab, the first Shelia Essey Award in ALS from the American Academy of Neurology, the Hope through Caring Award from the Les Turner ALS Foundation and the Forbes Norris Award from the International Alliance of ALS/MND Associations. Additionally, he was the inaugural recipient the Abbott Laboratories Duane and Susan Burnham Research Professorship at Northwestern. His research in neurogenetics and disease mechanisms of ALS has been generously funded by the National Institutes of Health since 1985.




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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