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Functional assessment of glucocerebrosidase modulator efficacy in primary patient-derived macrophages is essential for drug development and patient stratification.

Welsh, NJ; Gewinner, CA; Mistry, K; Koglin, M; Cooke, J; Butler, M; Powney, B; ... Schapira, AHV; + view all (2019) Functional assessment of glucocerebrosidase modulator efficacy in primary patient-derived macrophages is essential for drug development and patient stratification. Haematologica 10.3324/haematol.2019.224717. (In press). Green open access

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Type: Article
Title: Functional assessment of glucocerebrosidase modulator efficacy in primary patient-derived macrophages is essential for drug development and patient stratification.
Location: Italy
Open access status: An open access version is available from UCL Discovery
DOI: 10.3324/haematol.2019.224717
Publisher version: http://doi.org/10.3324/haematol.2019.224717
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Gaucher Disease, Granulocytes, Monocytes, Macrophages, Molecular Pharmacology, pharmacological chaperone
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Clinical and Movement Neurosciences
URI: https://discovery.ucl.ac.uk/id/eprint/10082992
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