Smita Saxena, PhD

Smita Saxena, PhD

Physical Medicine and Rehabilitation

NextGen Precision Health Investigator



The research program of Smita Saxena, PhD, is focused on mechanisms of neuronal vulnerability in the context of neurodegenerative diseases and aging. Employing state-of-the-art techniques, her lab examines fundamental principles ranging from circuits to molecular and cellular pathology that drive and propagate neuronal loss. Her lab is interested in identifying adaptive mechanisms, promoting resiliency of the nervous system during the presymptomatic phase and translating those mechanisms into potential therapy. Additionally, a major focus of her research involves the generation of human patient-derived neurons from skin fibroblasts, which in collaboration with the pharmaceutical/biotech industry are used as model systems to test future druggable compounds/repurposed drugs. Lastly, working with nanochemists and biomedical engineers, her lab has a keen interest in developing efficient brain targetable and prodrug approaches to enhance drug delivery to specialized brain regions.

NextGen Precision Health

Academic Information

NextGen Precision Health Investigator


1030 Hitt St
Roy Blunt NextGen Precision Health Building
Columbia, MO 65211
United States

P. 573-884-2924

Research Interests

  • Neural ensembles in pathological coding
  • Sensory motor integration deficits in ataxias
  • Neuronal engrams of neuroinflammation
  • Molecular correlates of circuit dysfunction
  • ER-mitochondria cross-talk in impaired calcium homeostasis
  • cGAS-STING pathway and the cell clearance machinery
  • Nano lipid pipeline for structured drug delivery

Areas of Expertise

  • Pathophysiological and cellular mechanisms of neurodegeneration.
  • Spinal and Cerebellar circuit modulation in preclinical rodent models
  • Orthogonal pharmacogenetics/chemogenetics
  • In vivo 2-photon imaging, optogenetics, and electrophysiology
  • High-resolution imaging and image analyses.
  • Neuron-specific OMICS, high throughput proteomics, and bioinformatic analyses
  • Direct human fibroblast and human iPSC-derived neurons and brain cells
  • 3D NMJ and human iPSC-derived organoid cultures, microfluidic assays
  • Human spinal cord and NMJ anatomical and cellular analyses
  • Viral-mediated gene therapy and tracing of neuronal circuits

Education & Training


2010 Postdoctoral Fellow, Friederich Miescher Institute for Biomedical Research, Basel
2004 PhD, University of Bern
2000 Masters, University of Lausanne


Post doctoral fellow, Group: Pico Caroni, Friedrich Miescher Institute, Basel, CH

Awards & Honors

  • European Research Council Consolidator Award, 2017-2022
  • Swiss National Science Foundation Professorship Award, 2010-2015
  • NeuroNE, Project Prize, 2007
  • NeuroNE, Project Prize, 2006
  • European Network, FP6, Network of Excellence postdoctoral support, 2005-2008


  • Pilotto F, Diab R, Al Qassab Z, Saxena S* (2024)
    Generation and Enrichment of Cerebellar GABAergic Interneurons from Human Induced Pluripotent Stem Cells and Intracellular Calcium Measurements.
    Star Protocols in press
  • Pilotto F, Douthwaite C, Diab R, Al Qassab Z, Ye XQ, Tietje C, Mounassir M, Odriozola A, Buijsen R.A.M, Lagache S, Uldry A.C, Heller M, Muller S, Thapa A, van Roon-Mom W, Liebscher S, Saxena S* (2023).
    Early molecular layer interneuron hyperactivity triggers Purkinje neuron degeneration in SCA1.
  • Diab R, Pilotto F, Saxena S* (2023)
    Evolving roles of C9orf72 in the regulation of autophagy and the relationship to ALS-FTD pathology.
    Front Cell Neurosci. doi: 10.3389/fncel.2023.1086895.
  • De Lorenzo F, Luningschror P, Nam J, Pilotto F, Galli E, Lindholm P, Rudt von Collenberg C, Mungwa ST, Kauder J, Jablonka S, Petri S, Lindholm D, Saxena S, Sendtner M, Saarma M, Voutilainen MH* (2023).
    CDNF rescues motor neurons in three animal models of ALS by targeting ER stress.
    Brain Mar 16:awad087 doi: 10.1093/brain/awad087.
  • Gomez Limia CE, Baird M, Schwartz M, Saxena S, Meyer KC, Wein N (2022)
    Emerging Perspectives on gene therapy delivery for Neurodegenerative and Neuromuscular Disorders.
    J. Pers. Med., 12(12), 1979;
  • Pilotto F, Maharjan N, Schmitz AJ, Diab R, Scheidegger O, Oestmann A, Odriozola A, Santo S.D, Dennys C.N, Sinha Ray S, Rodrigo R, Kolb S, Aronica E, Troost D, Tripathi P, Goswami A, Wies J, Charlet-Berguerand N, Widmer HR, Meyer K, Zuber B, Saxena S* (2022)
    PolyGA targets the ER stress-adaptive response by impairing GRP75 function at the MAM in C9ORF72-ALS.
    Acta Neuropathologica, Nov 144(5):939-966.
  • Schmitz A, Pinheiro Marques J, Oertig I, Maharjan N, Saxena S* (2021)
    Emerging Perspectives on Dipeptide Repeat Proteins in C9ORF72 ALS/FTD.
    Front Cell Neurosci. Feb 18;15:637548.