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Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
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- Dahl, Maria (author)
- Lund University,Lunds universitet,Hematopoes och genterapi,Forskargrupper vid Lunds universitet,Hematopoiesis and Gene Therapy,Lund University Research Groups
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- Smith, Emma M.K. (author)
- 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
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- Warsi, Sarah (author)
- Lund University,Lunds universitet,Hematopoes och genterapi,Forskargrupper vid Lunds universitet,Hematopoiesis and Gene Therapy,Lund University Research Groups
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- (creator_code:org_t)
- Elsevier BV, 2021
- 2021
- English 12 s.
- In: Molecular Therapy - Methods and Clinical Development. - : Elsevier BV. - 2329-0501. ; 20, s. 312-323
- Journal article (peer-reviewed)
Abstract
Subject headings
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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
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinska och farmaceutiska grundvetenskaper -- Cell- och molekylärbiologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Basic Medicine -- Cell and Molecular Biology (hsv//eng)
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- Dahl, Maria
- Smith, Emma M.K.
- Warsi, Sarah
- Rothe, Michael
- Ferraz, Maria J.
- Aerts, Johannes ...
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- Golipour, Azadeh
- Harper, Claudia
- Pfeifer, Richard
- Pizzurro, Daniel ...
- Schambach, Axel
- Mason, Chris
- Karlsson, Stefan
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