View in EDS HTML Full Text PDF Full Text

Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.

Saved in:
Bibliographic Details
Title: Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.
Authors: Sellier P; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France., Vidal P; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France., Bertin B; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France., Gicquel E; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France., Bertil-Froidevaux E; Genethon, Evry, France., Georger C; Genethon, Evry, France., van Wittenberghe L; Genethon, Evry, France., Miranda A; Genethon, Evry, France., Daniele N; Genethon, Evry, France., Richard I; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France., Gross DA; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France., Mingozzi F; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France., Collaud F; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France., Ronzitti G; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France.
Source: Journal of inherited metabolic disease [J Inherit Metab Dis] 2024 Jan; Vol. 47 (1), pp. 119-134. Date of Electronic Publication: 2023 May 29.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-2665 (Electronic) Linking ISSN: 01418955 NLM ISO Abbreviation: J Inherit Metab Dis 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: 37204237
AccessLevel: 2
PubType: Academic Journal
PubTypeId: academicJournal
PreciseRelevancyScore: 0
IllustrationInfo
Items – Name: Title
  Label: Title
  Group: Ti
  Data: Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa<superscript>-/-</superscript> mice.
– Name: Author
  Label: Authors
  Group: Au
  Data: <searchLink fieldCode="AU" term="%22Sellier+P%22">Sellier P</searchLink>; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Vidal+P%22">Vidal P</searchLink>; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Bertin+B%22">Bertin B</searchLink>; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Gicquel+E%22">Gicquel E</searchLink>; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Bertil-Froidevaux+E%22">Bertil-Froidevaux E</searchLink>; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Georger+C%22">Georger C</searchLink>; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22van+Wittenberghe+L%22">van Wittenberghe L</searchLink>; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Miranda+A%22">Miranda A</searchLink>; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Daniele+N%22">Daniele N</searchLink>; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Richard+I%22">Richard I</searchLink>; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Gross+DA%22">Gross DA</searchLink>; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Mingozzi+F%22">Mingozzi F</searchLink>; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Collaud+F%22">Collaud F</searchLink>; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France.<br /><searchLink fieldCode="AU" term="%22Ronzitti+G%22">Ronzitti G</searchLink>; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.; Genethon, Evry, France.
– Name: TitleSource
  Label: Source
  Group: Src
  Data: <searchLink fieldCode="JN" term="%227910918%22">Journal of inherited metabolic disease</searchLink> [J Inherit Metab Dis] 2024 Jan; Vol. 47 (1), pp. 119-134. <i>Date of Electronic Publication: </i>2023 May 29.
– Name: TypePub
  Label: Publication Type
  Group: TypPub
  Data: Journal Article; Research Support, Non-U.S. Gov't
– Name: TitleSource
  Label: Journal Info
  Group: Src
  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Wiley%22">Wiley </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>7910918 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1573-2665 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2201418955%22">01418955 </searchLink><i>NLM ISO Abbreviation: </i>J Inherit Metab Dis <i>Subsets: </i>MEDLINE
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=37204237
RecordInfo BibRecord:
  BibEntity:
    Identifiers:
      – Type: doi
        Value: 10.1002/jimd.12625
    Languages:
      – Code: eng
        Text: English
    PhysicalDescription:
      Pagination:
        StartPage: 119
    Titles:
      – TitleFull: Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.
        Type: main
  BibRelationships:
    HasContributorRelationships:
      – PersonEntity:
          Name:
            NameFull: Sellier P
      – PersonEntity:
          Name:
            NameFull: Vidal P
      – PersonEntity:
          Name:
            NameFull: Bertin B
      – PersonEntity:
          Name:
            NameFull: Gicquel E
      – PersonEntity:
          Name:
            NameFull: Bertil-Froidevaux E
      – PersonEntity:
          Name:
            NameFull: Georger C
      – PersonEntity:
          Name:
            NameFull: van Wittenberghe L
      – PersonEntity:
          Name:
            NameFull: Miranda A
      – PersonEntity:
          Name:
            NameFull: Daniele N
      – PersonEntity:
          Name:
            NameFull: Richard I
      – PersonEntity:
          Name:
            NameFull: Gross DA
      – PersonEntity:
          Name:
            NameFull: Mingozzi F
      – PersonEntity:
          Name:
            NameFull: Collaud F
      – PersonEntity:
          Name:
            NameFull: Ronzitti G
    IsPartOfRelationships:
      – BibEntity:
          Dates:
            – D: 01
              M: 01
              Text: 2024 Jan
              Type: published
              Y: 2024
          Identifiers:
            – Type: issn-electronic
              Value: 1573-2665
          Numbering:
            – Type: volume
              Value: 47
            – Type: issue
              Value: 1
          Titles:
            – TitleFull: Journal of inherited metabolic disease
              Type: main
ResultId 1