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Smita Saxena, PhD

Smita Saxena, Ph.D. portrait

Professor of Physical Medicine and Rehabilitation, School of Medicine

NextGen Focus Area: Neurodegenerative diseases, neuromuscular and neurodevelopmental disorders, and therapy

Dr. Saxena’s main research aim is to identify cellular mechanisms causally involved with the onset and spreading of neurodegenerative diseases. In her work, she uses cutting-edge imaging and human cellular models combined with systems biology to identify and investigate disease-related aberrant cellular mechanisms, which form the framework for future novel therapies.


Precision Health Impact:

  • Improved understanding of the disease process
  • Identification of valid biomarkers for disease staging and diagnosis
  • Novel druggable targets


De Lorenzo F, Lüningschrör P, Nam J, Beckett L, Pilotto F, Galli E, et al. CDNF rescues motor neurons in models of amyotrophic lateral sclerosis by targeting endoplasmic reticulum stress. Brain. 2023 Sep 1;146(9):3783–99.

Pilotto F, Douthwaite C, Diab R, Ye X, Al Qassab Z, Tietje C, et al. Early molecular layer interneuron hyperactivity triggers Purkinje neuron degeneration in SCA1. Neuron. 2023 Aug 16;111(16):2523-2543.e10.

Ruegsegger C, Stucki DM, Steiner S, Angliker N, Radecke J, Keller E, et al. Impaired mTORC1-Dependent Expression of Homer-3 Influences SCA1 Pathophysiology. Neuron. 2016 Jan 6;89(1):129–46.

Filézac de L’Etang A, Maharjan N, Cordeiro Braña M, Ruegsegger C, Rehmann R, Goswami A, et al. Marinesco-Sjögren syndrome protein SIL1 regulates motor neuron subtype-selective ER stress in ALS. Nat Neurosci. 2015 Feb;18(2):227–38.

Pilotto F, Schmitz A, Maharjan N, Diab R, Odriozola A, Tripathi P, et al. PolyGA targets the ER stress-adaptive response by impairing GRP75 function at the MAM in C9ORF72-ALS/FTD. Acta Neuropathol. 2022 Nov;144(5):939–66.
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