Justin Ichida, PhD

Associate Professor of Stem Cell Biology and Regenerative Medicine

John Douglas French Alzheimer’s Endowed Professor in Regenerative Medicine

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Overview

Ichida Lab Website

Research Funding

  • Leveraging Natural Phenotypic Variations of Heterogenous ALS Populations-in-a-Dish to Enable Scalable Drug Discovery
    NIH · R01NS131409 · Sep 18, 2022 – Aug 31, 2027 · Role: Principal Investigator
  • Validating Secretory Autophagy as a Therapeutic Strategy for Diverse Forms of ALS and FTD
    NIH · R01NS097850 · Sep 1, 2016 – Jun 30, 2025 · Role: Principal Investigator
  • Lineage reprogramming for hearing loss: development of drug screening and gene therapy approaches
    NIH · R01DC015530 · Jul 1, 2016 – Jun 30, 2021 · Role: Co-Principal Investigator
  • Conversion of Fibroblasts to fxnal Spinal Motor Neurons Using Defined Factor
    NIH · R00NS077435 · Jan 1, 2013 – Dec 31, 2016 · Role: Principal Investigator
  • Conversion of fibroblasts to functional spinal motor neurons using defined factor
    NIH · K99NS077435 · Sep 30, 2011 – Aug 31, 2013 · Role: Principal Investigator

Publications

  • C9orf72 -associated G4C2 hexanucleotide repeat expression in Drosophila mushroom bodies causes age dependent TDP-43 pathology and dementia relevant phenotypes mediated in part by the glypican Dlp/GPC6. bioRxiv. 2026 May 12.. View in PubMed
  • Unveiling alternate pathways for SARS-CoV-2 infection via extracellular vesicle-mediated transfer of ACE2 and TMPRSS2. Nat Commun. 2026 Apr 10; 17(1).. View in PubMed
  • C9orf72 hexanucleotide repeat RNA drives transcriptional dysregulation through genome-wide DNA:RNA hybrid G-quadruplexes. Neuron. 2026 Mar 18; 114(6):1045-1065.e13.. View in PubMed
  • PIKFYVE inhibition mitigates disease in models of diverse forms of ALS. Cell. 2026 Feb 05; 189(3):987-992.. View in PubMed
  • The Ku80-p53-SIRT1 axis in DNA damage response contributes to sporadic and familial ALS and FTD. Nat Commun. 2025 Dec 20; 17(1):1007.. View in PubMed
  • AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders. Mol Ther. 2025 Dec 03; 33(12):6398-6414.. View in PubMed
  • AAV-mediated exon skipping therapy for Usher syndrome, type 2A. Mol Ther. 2026 Jan 07; 34(1):179-202.. View in PubMed
  • Spatially patterned kidney assembloids recapitulate progenitor self-assembly and enable high-fidelity in vivo disease modeling. Cell Stem Cell. 2025 Oct 02; 32(10):1614-1633.e13.. View in PubMed
  • Elevated tripeptidyl-peptidase 1 corrects multiple disease phenotypes in a mouse model of juvenile neuronal ceroid lipofuscinosis. Mol Ther Methods Clin Dev. 2025 Dec 11; 33(4):101587.. View in PubMed
  • Antisense oligonucleotide depletion of CCDC146 is a broad-spectrum therapeutic strategy for ALS. medRxiv. 2025 Aug 19.. View in PubMed