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

doi:10.1016/j.omtm.2020.11.018

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

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Abstract

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.

Original language	English
Pages (from-to)	312-323
Number of pages	12
Journal	Molecular Therapy - Methods and Clinical Development
Volume	20
DOIs	https://doi.org/10.1016/j.omtm.2020.11.018
Publication status	Published - 2021

Subject classification (UKÄ)

Cell and Molecular Biology

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10.1016/j.omtm.2020.11.018

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Dahl, M., Smith, E. M. K., Warsi, S., Rothe, M., Ferraz, M. J., Aerts, J. M. F. G., Golipour, A., Harper, C., Pfeifer, R., Pizzurro, D., Schambach, A., Mason, C., & Karlsson, S. (2021). Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector. Molecular Therapy - Methods and Clinical Development, 20, 312-323. https://doi.org/10.1016/j.omtm.2020.11.018

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

abstract = "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.",

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

year = "2021",

doi = "10.1016/j.omtm.2020.11.018",

language = "English",

volume = "20",

pages = "312--323",

journal = "Molecular Therapy - Methods and Clinical Development",

issn = "2329-0501",

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Dahl, M, Smith, EMK, Warsi, S, Rothe, M, Ferraz, MJ, Aerts, JMFG, Golipour, A, Harper, C, Pfeifer, R, Pizzurro, D, Schambach, A, Mason, C & Karlsson, S 2021, 'Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector', Molecular Therapy - Methods and Clinical Development, vol. 20, pp. 312-323. https://doi.org/10.1016/j.omtm.2020.11.018

TY - JOUR

T1 - Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector

AU - Dahl, Maria

AU - Smith, Emma M.K.

AU - Warsi, Sarah

AU - Rothe, Michael

AU - Ferraz, Maria J.

AU - Aerts, Johannes M.F.G.

AU - Golipour, Azadeh

AU - Harper, Claudia

AU - Pfeifer, Richard

AU - Pizzurro, Daniella

AU - Schambach, Axel

AU - Mason, Chris

AU - Karlsson, Stefan

PY - 2021

Y1 - 2021

N2 - 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.

AB - 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.

UR - https://www.scopus.com/pages/publications/85099244856

U2 - 10.1016/j.omtm.2020.11.018

DO - 10.1016/j.omtm.2020.11.018

M3 - Article

C2 - 33511245

AN - SCOPUS:85099244856

SN - 2329-0501

VL - 20

SP - 312

EP - 323

JO - Molecular Therapy - Methods and Clinical Development

JF - Molecular Therapy - Methods and Clinical Development

ER -

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

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