Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model.
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| Title: | Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model. |
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| Authors: | Wan F; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA., He J; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA., Ma H; Viral Vector Core, University of Massachusetts Chan Medical School, Worcester, MA, USA., PiresFerreira D; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA., Kumanan V; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA., Lee JS; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.; Viral Vector Core, University of Massachusetts Chan Medical School, Worcester, MA, USA., Chen X; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA., He R; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.; Viral Vector Core, University of Massachusetts Chan Medical School, Worcester, MA, USA., Su Q; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.; Viral Vector Core, University of Massachusetts Chan Medical School, Worcester, MA, USA., Gallagher TL; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA., Zhu S; Neushen Therapeutics US Inc., Burlington, MA, USA., Cabrera GT; Neushen Therapeutics US Inc., Burlington, MA, USA., Zhao L; Neushen Therapeutics US Inc., Burlington, MA, USA., Shen J; Neushen Therapeutics US Inc., Burlington, MA, USA., Gruntman A; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA., Brown RH Jr; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, USA., Xu Z; Department of Biochemistry & Molecular Biotechnology, University of Massachusetts Chan Medical School, Worcester, MA, USA. zuoshang.xu@umassmed.edu., Gao G; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA. guangping.gao@umassmed.edu.; Viral Vector Core, University of Massachusetts Chan Medical School, Worcester, MA, USA. guangping.gao@umassmed.edu.; Department of Microbiology, University of Massachusetts Chan Medical School, Worcester, MA, USA. guangping.gao@umassmed.edu., Xie J; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA. jun.xie@umassmed.edu.; Viral Vector Core, University of Massachusetts Chan Medical School, Worcester, MA, USA. jun.xie@umassmed.edu.; Department of Microbiology, University of Massachusetts Chan Medical School, Worcester, MA, USA. jun.xie@umassmed.edu. |
| Source: | Nature communications [Nat Commun] 2026 Jun 25; Vol. 17 (1). Date of Electronic Publication: 2026 Jun 25. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 2041-1723 |
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| DOI: | 10.1038/s41467-026-74169-8 |
