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

Maria Dahl, Emma M.K. Smith, Sarah Warsi, Michael Rothe, Maria J. Ferraz, Johannes M.F.G. Aerts, Azadeh Golipour, Claudia Harper, Richard Pfeifer, Daniella Pizzurro, Axel Schambach, Chris Mason, Stefan Karlsson

Forskningsoutput: TidskriftsbidragArtikel i vetenskaplig tidskriftPeer review

4 Citeringar (SciVal)

Sammanfattning

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.

Originalspråkengelska
Sidor (från-till)312-323
Antal sidor12
TidskriftMolecular Therapy - Methods and Clinical Development
Volym20
DOI
StatusPublished - 2021

Ämnesklassifikation (UKÄ)

  • Cell- och molekylärbiologi

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