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Recent Publications

  1. Banach-Petrosky, W, Larrimore, KE, Sleat, EH, Bazer, A, Samuels, B, Tan, Y et al.. Elevated tripeptidyl-peptidase 1 corrects multiple disease phenotypes in a mouse model of juvenile neuronal ceroid lipofuscinosis. Mol Ther Methods Clin Dev. 2025;33 (4):101587. doi: 10.1016/j.omtm.2025.101587. PubMed PMID:41049692 PubMed Central PMC12493141.
  2. Mauriac, SA, Lee, J, Zhang, J, Jin, J, Nist-Lund, C, Martin, CT et al.. AAV-Mediated Exon Skipping Therapy for Usher Syndrome, Type 2A. Mol Ther. 2025; :. doi: 10.1016/j.ymthe.2025.09.038. PubMed PMID:40994011 .
  3. Quinlan, MA, Guo, R, Clark, AG, Luber, EM, Christian, RJ, Martinez, RA et al.. AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders. Mol Ther. 2025; :. doi: 10.1016/j.ymthe.2025.09.040. PubMed PMID:40988338 .
  4. Huang, B, Medina, P, He, J, Zeng, Z, Kim, S, Romo, J et al.. Spatially patterned kidney assembloids recapitulate progenitor self-assembly and enable high-fidelity in vivo disease modeling. Cell Stem Cell. 2025;32 (10):1614-1633.e13. doi: 10.1016/j.stem.2025.08.013. PubMed PMID:40967224 .
  5. Zhang, S, Shu, H, Zhou, J, Rubin-Sigler, J, Yang, X, Liu, Y et al.. Single-cell polygenic risk scores dissect cellular and molecular heterogeneity of complex human diseases. Nat Biotechnol. 2025; :. doi: 10.1038/s41587-025-02725-6. PubMed PMID:40715455 .
  6. Yu, SF, Michon, M, Lingappa, AF, Paulvannan, K, Solas, D, Staats, K et al.. An ALS assembly modulator signature in peripheral blood mononuclear cells: implications for ALS pathophysiology, therapeutics, and diagnostics. Clin Proteomics. 2025;22 (1):16. doi: 10.1186/s12014-025-09538-4. PubMed PMID:40295933 PubMed Central PMC12036218.
  7. Salomon-Zimri, S, Kerem, N, Linares, GR, Russek-Blum, N, Ichida, JK, Tracik, F et al.. Elucidating the Synergistic Effect of the PrimeC Combination for Amyotrophic Lateral Sclerosis in Human Induced Pluripotent Stem Cell-Derived Motor Neurons and Mouse Models. Pharmaceuticals (Basel). 2025;18 (4):. doi: 10.3390/ph18040524. PubMed PMID:40283960 PubMed Central PMC12030000.
  8. Berlind, JE, Lai, JD, Lie, C, Vicente, J, Lam, K, Guo, S et al.. KCTD20 suppression mitigates excitotoxicity in tauopathy patient organoids. Neuron. 2025;113 (8):1169-1189.e7. doi: 10.1016/j.neuron.2025.02.001. PubMed PMID:40049159 PubMed Central PMC12005969.
  9. Santoso, JW, Do, SK, Verma, R, Do, AV, Hendricks, E, Ichida, JK et al.. Human iPSC-Derived Motor Neuron Innervation Enhances the Differentiation of Muscle Bundles Engineered with Benchtop Fabrication Techniques. ACS Biomater Sci Eng. 2025;11 (3):1731-1740. doi: 10.1021/acsbiomaterials.4c02225. PubMed PMID:39973396 PubMed Central PMC11897949.
  10. Santoso, JW, Do, SK, Verma, R, Do, AV, Hendricks, E, Ichida, JK et al.. Human iPSC-derived motor neuron innervation enhances the differentiation of muscle bundles engineered with benchtop fabrication techniques. bioRxiv. 2024; :. doi: 10.1101/2024.12.02.626391. PubMed PMID:39677637 PubMed Central PMC11642770.