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  • Dahl, MariaLund University,Lunds universitet,Hematopoes och genterapi,Forskargrupper vid Lunds universitet,Hematopoiesis and Gene Therapy,Lund University Research Groups (author)

Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector

  • Article/chapterEnglish2021

Publisher, publication year, extent ...

  • Elsevier BV,2021
  • 12 s.

Numbers

  • LIBRIS-ID:oai:lup.lub.lu.se:1b83addf-4c37-44a8-b753-b0ce9bd5daf7
  • https://lup.lub.lu.se/record/1b83addf-4c37-44a8-b753-b0ce9bd5daf7URI
  • https://doi.org/10.1016/j.omtm.2020.11.018DOI

Supplementary language notes

  • Language:English
  • Summary in:English

Part of subdatabase

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  • Subject category:art swepub-publicationtype
  • Subject category:ref swepub-contenttype

Notes

  • This study evaluates a clinically applicable lentiviral vector for treatment of Gaucher disease type 1. Hematopoietic stem cells transduced with the vector and transplanted into a mouse model successfully halted or reversed pathology. These data were used as proof-of-concept for regulatory filing enabling the commencement of an international phase 1/2 clinical trial.

Subject headings and genre

Added entries (persons, corporate bodies, meetings, titles ...)

  • Smith, Emma M.K.Lund University,Lunds universitet,Avdelningen för molekylär hematologi,Institutionen för laboratoriemedicin,Medicinska fakulteten,Division of Molecular Hematology (DMH),Department of Laboratory Medicine,Faculty of Medicine(Swepub:lu)mphy-esm (author)
  • Warsi, SarahLund University,Lunds universitet,Hematopoes och genterapi,Forskargrupper vid Lunds universitet,Hematopoiesis and Gene Therapy,Lund University Research Groups(Swepub:lu)med-swr (author)
  • Rothe, MichaelHannover Medical School (author)
  • Ferraz, Maria J.Leiden University (author)
  • Aerts, Johannes M.F.G.Leiden University (author)
  • Golipour, AzadehAvrobio Inc (author)
  • Harper, ClaudiaAvrobio Inc (author)
  • Pfeifer, RichardAvrobio Inc (author)
  • Pizzurro, DaniellaAvrobio Inc (author)
  • Schambach, AxelHannover Medical School (author)
  • Mason, ChrisAvrobio Inc,University College London (author)
  • Karlsson, StefanLund University,Lunds universitet,Hematopoes och genterapi,Forskargrupper vid Lunds universitet,Hematopoiesis and Gene Therapy,Lund University Research Groups(Swepub:lu)molm-ska (author)
  • Hematopoes och genterapiForskargrupper vid Lunds universitet (creator_code:org_t)

Related titles

  • In:Molecular Therapy - Methods and Clinical Development: Elsevier BV20, s. 312-3232329-0501

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