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Correction of patho...
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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- Rothe, Michael (author)
- Hannover Medical School
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- Ferraz, Maria J. (author)
- Leiden University
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- Aerts, Johannes M.F.G. (author)
- Leiden University
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- Golipour, Azadeh (author)
- Avrobio Inc
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- Harper, Claudia (author)
- Avrobio Inc
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- Pfeifer, Richard (author)
- Avrobio Inc
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- Pizzurro, Daniella (author)
- Avrobio Inc
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- Schambach, Axel (author)
- Hannover Medical School
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- Mason, Chris (author)
- Avrobio Inc,University College London
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- Karlsson, Stefan (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.
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In: Molecular Therapy - Methods and Clinical Development. - : Elsevier BV. - 2329-0501. ; 20, s. 312-323
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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)
Publication and Content Type
- art (subject category)
- ref (subject category)
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- By the author/editor
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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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show more...
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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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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Basic Medicine
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and Cell and Molecul ...
- Articles in the publication
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Molecular Therap ...
- By the university
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Lund University