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Correction of patho...
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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 ...
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LIBRIS-ID:oai:lup.lub.lu.se:1b83addf-4c37-44a8-b753-b0ce9bd5daf7
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https://lup.lub.lu.se/record/1b83addf-4c37-44a8-b753-b0ce9bd5daf7URI
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https://doi.org/10.1016/j.omtm.2020.11.018DOI
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Language:English
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Summary in:English
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Subject category:art swepub-publicationtype
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Subject category:ref swepub-contenttype
Notes
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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 ...)
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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)
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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)
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Rothe, MichaelHannover Medical School
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Ferraz, Maria J.Leiden University
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Aerts, Johannes M.F.G.Leiden University
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Golipour, AzadehAvrobio Inc
(author)
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Harper, ClaudiaAvrobio Inc
(author)
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Pfeifer, RichardAvrobio Inc
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Pizzurro, DaniellaAvrobio Inc
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Schambach, AxelHannover Medical School
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Mason, ChrisAvrobio Inc,University College London
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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)
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Hematopoes och genterapiForskargrupper vid Lunds universitet
(creator_code:org_t)
Related titles
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In:Molecular Therapy - Methods and Clinical Development: Elsevier BV20, s. 312-3232329-0501
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Dahl, Maria
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Smith, Emma M.K.
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Warsi, Sarah
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Rothe, Michael
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Ferraz, Maria J.
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Aerts, Johannes ...
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Golipour, Azadeh
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Harper, Claudia
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Pfeifer, Richard
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Pizzurro, Daniel ...
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Schambach, Axel
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Mason, Chris
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Karlsson, Stefan
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- About the subject
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- MEDICAL AND HEALTH SCIENCES
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Molecular Therap ...
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Lund University