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Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model

Crossref DOI link: https://doi.org/10.1038/s41467-026-74169-8

Published Online: 2026-06-25

Update policy: https://doi.org/10.1007/springer_crossmark_policy

Authors

Wan, Fang

He, Jinchen

Ma, Hong

PiresFerreira, Debora https://orcid.org/0000-0001-7747-9599
Kumanan, Veena

Lee, Ji Sun

Chen, Xiupeng https://orcid.org/0000-0001-5916-3953
He, Ran

Su, Qin

Gallagher, Thomas L. https://orcid.org/0000-0001-6381-6902
Zhu, Sha

Cabrera, Gabriela Toro https://orcid.org/0000-0002-8905-1589
Zhao, Lingzhi

Shen, Joan

Gruntman, Alisha

Brown, Robert H. Jr https://orcid.org/0000-0001-6062-1528
Xu, Zuoshang https://orcid.org/0000-0003-1209-334X
Gao, Guangping https://orcid.org/0000-0003-0097-9012
Xie, Jun https://orcid.org/0000-0001-9565-1567
Funding

Funding for this research was provided by:

United States Department of Defense | United States Army | Army Medical Command | Congressionally Directed Medical Research Programs (AL240123)
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Text and Data Mining valid from 2026-06-25

Version of Record valid from 2026-06-25
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Article History

Received: 5 August 2025

Accepted: 28 May 2026

First Online: 25 June 2026

Competing interests

: F.W., H.M., Z.X., G.G., and J.X. are inventors of a patent application filed by the UMass Chan Medical School on SMA gene therapy. G.G. is a scientific co-founder of Voyager Therapeutics, Adrenas Therapeutics, AAVAA Therapeutics, and Aspa Therapeutics and holds equity in these companies. G.G. and J.X. are inventors on patents related to AAV-based gene therapy, some of which have been licensed to commercial entities. S.Z., L.Z., G.C., and J.S. are employees or former employees of NeuShen Therapeutics US, Inc. All other authors declare no competing interests.

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