View in EDS HTML Full Text PDF Full Text

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

Saved in:
Bibliographic Details
Title: Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model.
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
Full text is not displayed to guests.
FullText Links:
  – Type: pdflink
Text:
  Availability: 1
Header DbId: mdl
DbLabel: MEDLINE Ultimate
An: 42350385
AccessLevel: 2
PubType: Academic Journal
PubTypeId: academicJournal
PreciseRelevancyScore: 0
IllustrationInfo
Items – Name: Title
  Label: Title
  Group: Ti
  Data: Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model.
– Name: Author
  Label: Authors
  Group: Au
  Data: <searchLink fieldCode="AU" term="%22Wan+F%22">Wan F</searchLink>; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22He+J%22">He J</searchLink>; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Ma+H%22">Ma H</searchLink>; Viral Vector Core, University of Massachusetts Chan Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22PiresFerreira+D%22">PiresFerreira D</searchLink>; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Kumanan+V%22">Kumanan V</searchLink>; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Lee+JS%22">Lee JS</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Chen+X%22">Chen X</searchLink>; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22He+R%22">He R</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Su+Q%22">Su Q</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Gallagher+TL%22">Gallagher TL</searchLink>; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Zhu+S%22">Zhu S</searchLink>; Neushen Therapeutics US Inc., Burlington, MA, USA.<br /><searchLink fieldCode="AU" term="%22Cabrera+GT%22">Cabrera GT</searchLink>; Neushen Therapeutics US Inc., Burlington, MA, USA.<br /><searchLink fieldCode="AU" term="%22Zhao+L%22">Zhao L</searchLink>; Neushen Therapeutics US Inc., Burlington, MA, USA.<br /><searchLink fieldCode="AU" term="%22Shen+J%22">Shen J</searchLink>; Neushen Therapeutics US Inc., Burlington, MA, USA.<br /><searchLink fieldCode="AU" term="%22Gruntman+A%22">Gruntman A</searchLink>; Horae Gene Therapy Center, Department of Genetic and Cellular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Brown+RH+Jr%22">Brown RH Jr</searchLink>; Department of Neurology, University of Massachusetts Chan Medical School, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Xu+Z%22">Xu Z</searchLink>; Department of Biochemistry & Molecular Biotechnology, University of Massachusetts Chan Medical School, Worcester, MA, USA. zuoshang.xu@umassmed.edu.<br /><searchLink fieldCode="AU" term="%22Gao+G%22">Gao G</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Xie+J%22">Xie J</searchLink>; 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.
– Name: TitleSource
  Label: Source
  Group: Src
  Data: <searchLink fieldCode="JN" term="%22101528555%22">Nature communications</searchLink> [Nat Commun] 2026 Jun 25; Vol. 17 (1). <i>Date of Electronic Publication: </i>2026 Jun 25.
– Name: TypePub
  Label: Publication Type
  Group: TypPub
  Data: Journal Article
– Name: TitleSource
  Label: Journal Info
  Group: Src
  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Nature+Pub%2E+Group%22">Nature Pub. Group </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>101528555 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2041-1723 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2220411723%22">20411723 </searchLink><i>NLM ISO Abbreviation: </i>Nat Commun <i>Subsets: </i>MEDLINE
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=42350385
RecordInfo BibRecord:
  BibEntity:
    Identifiers:
      – Type: doi
        Value: 10.1038/s41467-026-74169-8
    Languages:
      – Code: eng
        Text: English
    Titles:
      – TitleFull: Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model.
        Type: main
  BibRelationships:
    HasContributorRelationships:
      – PersonEntity:
          Name:
            NameFull: Wan F
      – PersonEntity:
          Name:
            NameFull: He J
      – PersonEntity:
          Name:
            NameFull: Ma H
      – PersonEntity:
          Name:
            NameFull: PiresFerreira D
      – PersonEntity:
          Name:
            NameFull: Kumanan V
      – PersonEntity:
          Name:
            NameFull: Lee JS
      – PersonEntity:
          Name:
            NameFull: Chen X
      – PersonEntity:
          Name:
            NameFull: He R
      – PersonEntity:
          Name:
            NameFull: Su Q
      – PersonEntity:
          Name:
            NameFull: Gallagher TL
      – PersonEntity:
          Name:
            NameFull: Zhu S
      – PersonEntity:
          Name:
            NameFull: Cabrera GT
      – PersonEntity:
          Name:
            NameFull: Zhao L
      – PersonEntity:
          Name:
            NameFull: Shen J
      – PersonEntity:
          Name:
            NameFull: Gruntman A
      – PersonEntity:
          Name:
            NameFull: Brown RH Jr
      – PersonEntity:
          Name:
            NameFull: Xu Z
      – PersonEntity:
          Name:
            NameFull: Gao G
      – PersonEntity:
          Name:
            NameFull: Xie J
    IsPartOfRelationships:
      – BibEntity:
          Dates:
            – D: 25
              M: 06
              Text: 2026 Jun 25
              Type: published
              Y: 2026
          Identifiers:
            – Type: issn-electronic
              Value: 2041-1723
          Numbering:
            – Type: volume
              Value: 17
            – Type: issue
              Value: 1
          Titles:
            – TitleFull: Nature communications
              Type: main
ResultId 1