OSCR

NeuroD1 gene therapy inhibits glioma growth and extends life span through <i>in vivo</i> reprogramming approach.

Overview

Authors: Yuchen Chen1, Zuoyu Jiang2, Sen Jin1, Meng Liu1, Ming Chen1, Tsang-Chih Kuo1, Kai Zhou1, Liting Pu1, Ming Chen1, Shiyuan Chen1, Xuetao Li2, Adalia S Chen1, Jingmu Xie3, Huitao Zhang3, Qingsong Wang3,4, Jie Xu1, Jian Sheng1, Yulun Huang2, Gong Chen1,3
ORCID iDs: Gong Chen
  1. NeuExcell Therapeutics, 218 Xinghu Street, Building B1, Floor 9, Suzhou, Jiangsu 215000, China
  2. Department of Neurosurgery, The Fourth Affiliated Hospital of Soochow University, Suzhou 215000, China
  3. Guangdong-Hong Kong-Macau Institute of CNS Regeneration (GHMICR), Jinan University, Guangzhou 510632, China
  4. Chinese Medicine Guangdong Laboratory, Hengqin, Guangdong 519000, China
Institutions: The Fourth Affiliated Hospital of Soochow University (Suzhou Dushu Lake Hospital); Jinan University (China)
Journal: Molecular therapy. Oncology, volume 34, issue 2, article 201205
Dates: received 14 May 2025; accepted 9 April 2026; published online 13 April 2026
Type: Research article · Language: English
License: CC BY
Identifiers: DOI 10.1016/j.omton.2026.201205 · PMID 42088619 · PMCID PMC13136644 · OpenAlex W7154083596
Open access: gold, a free copy (OpenAlex)
Status: code on request
Categories: mouse (organism), other condition (population)
Methods: Statistics, Evoked potentials
Keywords: glioblastoma, GBM, gene therapy, NeuroD1, AAV, tumor reprogramming, survival extension
Topic: Nerve injury and regeneration (Cellular and Molecular Neuroscience, Neuroscience), according to OpenAlex
Funding: National Natural Science Foundation of China
Citations: not cited yet (Europe PMC); 43 references in the paper

Abstract

Glioblastoma(GBM), a highly aggressive primary brain tumor characterized by rapid progression, frequent recurrence, and limited clinical options, remains one of the most lethal central nervous system malignancies. Here, we report a gene therapy strategy to treat glioma utilizing NeuroD1, a neurogenic transcription factor with demonstrated capacity to reprogram both glial cells and GBM cells into neuronal lineages. We developed a self-complementary adeno-associated virus (scAAV) vector, scAAV6-NeuroD1, and evaluated its therapeutic potential across in vitro and in vivo GBM models, including multiple GBM cell lines, patient-derived organoids, and orthotopic models in immunodeficient mice. Our findings reveal that scAAV6-NeuroD1 preferentially infects glioma cells and induces dual therapeutic effects by simultaneously inhibiting glioma cell proliferation and inducing neuronal reprogramming. Importantly, scAAV6-NeuroD1-treated mice with orthotopic GBM transplants exhibited reduced tumor burden, infiltration of T cells into the glioma, attenuated tumor-induced body weight loss, and dose-dependent survival extension. Analysis of published patient datasets further revealed that high NeuroD1 expression level correlates with improved overall survival and lower tumor malignancy grade. Together, these findings position scAAV6-NeuroD1 as a promising therapeutic candidate, potentially redefining the therapeutic landscape for GBM.

Reproduced under the paper's license (CC BY), from the paper cited above.

Code

The paper says that its authors' code is available on request: it was not published with the paper, so there is nothing to verify.

The paper's code and data availability statement is in the Data section.

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Data

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Data and code availability

The data that support the findings of this study are available from the corresponding author upon reasonable request.

Reproduced under the paper's license (CC BY), from the paper cited above.

Versions

The history of this record: each version stored by the harvester or made by a correction of its authors or of the maintainers of its code, and what changed in its facts. The texts of the paper (its abstract, its availability statements) are not part of it; versions that changed only those are not listed.

Version 1, 29 September 2026: the first record

Recorded: type, language, journal, volume, issue, pages, dates, 19 authors, 7 keywords, 1 funder, 42 references.

Cite

This paper

Chen, Y., Jiang, Z., Jin, S., Liu, M., Chen, M., Kuo, T.-C., Zhou, K., Pu, L., Chen, M., Chen, S., Li, X., Chen, A. S., Xie, J., Zhang, H., Wang, Q., Xu, J., Sheng, J., Huang, Y., & Chen, G. (2026). NeuroD1 gene therapy inhibits glioma growth and extends life span through <i>in vivo</i> reprogramming approach. Molecular therapy. Oncology, 34(2), 201205. https://doi.org/10.1016/j.omton.2026.201205

BibTeX

@article{chen2026neurod1,
author = {Chen, Yuchen and Jiang, Zuoyu and Jin, Sen and Liu, Meng and Chen, Ming and Kuo, Tsang-Chih and Zhou, Kai and Pu, Liting and Chen, Ming and Chen, Shiyuan and Li, Xuetao and Chen, Adalia S and Xie, Jingmu and Zhang, Huitao and Wang, Qingsong and Xu, Jie and Sheng, Jian and Huang, Yulun and Chen, Gong},
title = {{NeuroD1 gene therapy inhibits glioma growth and extends life span through \<i\>in vivo\</i\> reprogramming approach}},
journal = {Molecular therapy. Oncology},
year = {2026},
month = apr,
volume = {34},
number = {2},
pages = {201205},
publisher = {American Society of Gene \& Cell Therapy},
issn = {2950-3299},
doi = {10.1016/j.omton.2026.201205},
url = {https://doi.org/10.1016/j.omton.2026.201205},
pmid = {42088619},
pmcid = {PMC13136644}
}

RIS

TY - JOUR
AU - Chen, Yuchen
AU - Jiang, Zuoyu
AU - Jin, Sen
AU - Liu, Meng
AU - Chen, Ming
AU - Kuo, Tsang-Chih
AU - Zhou, Kai
AU - Pu, Liting
AU - Chen, Ming
AU - Chen, Shiyuan
AU - Li, Xuetao
AU - Chen, Adalia S
AU - Xie, Jingmu
AU - Zhang, Huitao
AU - Wang, Qingsong
AU - Xu, Jie
AU - Sheng, Jian
AU - Huang, Yulun
AU - Chen, Gong
TI - NeuroD1 gene therapy inhibits glioma growth and extends life span through <i>in vivo</i> reprogramming approach
T2 - Molecular therapy. Oncology
J2 - Mol Ther Oncol
PY - 2026
DA - 2026/04/13
VL - 34
IS - 2
SP - 201205
SN - 2950-3299
PB - American Society of Gene & Cell Therapy
DO - 10.1016/j.omton.2026.201205
UR - https://doi.org/10.1016/j.omton.2026.201205
LA - en
ER -

CSL-JSON

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