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Expert Opinion on Therapeutic Targets Volume 19, 2015 - Issue 3
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Review

Emerging therapeutic targets for Gaucher disease

Einat B VitnerWeizmann Institute of Science, Department of Biological Chemistry, Rehovot 76100, Israel+972 8 9342353; +972 8 9344112; [email protected].
,
Ayelet VardiWeizmann Institute of Science, Department of Biological Chemistry, Rehovot 76100, Israel+972 8 9342353; +972 8 9344112; [email protected].
,
Timothy M CoxUniversity of Cambridge, Department of Medicine, Cambridge CB2 0QQ, UK
&
Anthony H FutermanWeizmann Institute of Science, Department of Biological Chemistry, Rehovot 76100, Israel+972 8 9342353; +972 8 9344112; [email protected].
Pages 321-334 | Published online: 21 Nov 2014

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Jenny Kim, Ellen Sidransky & Grisel Lopez. (2015) Understanding and managing parkinsonism in patients with glucocerebrosidase mutations. Expert Opinion on Orphan Drugs 3:5, pages 549-562.
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Manoj Kumar Pandey. (2023) Exploring Pro-Inflammatory Immunological Mediators: Unraveling the Mechanisms of Neuroinflammation in Lysosomal Storage Diseases. Biomedicines 11:4, pages 1067.
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Batoul M. Issleny, Rama Jamjoum, Saurav Majumder & Johnny Stiban. 2023. History of The Enzymes, Current Topics and Future Perspectives. History of The Enzymes, Current Topics and Future Perspectives 171 201 .
Chandra Sekhar Boddupalli, Shiny Nair, Glenn Belinsky, Joseph Gans, Erin Teeple, Tri-Hung Nguyen, Sameet Mehta, Lilu Guo, Martin L Kramer, Jiapeng Ruan, Honggge Wang, Matthew Davison, Dinesh Kumar, DJ Vidyadhara, Bailin Zhang, Katherine Klinger & Pramod K Mistry. (2022) Neuroinflammation in neuronopathic Gaucher disease: Role of microglia and NK cells, biomarkers, and response to substrate reduction therapy. eLife 11.
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Laetitia Francelle & Joseph R. Mazzulli. (2022) Neuroinflammation in Gaucher disease, neuronal ceroid lipofuscinosis, and commonalities with Parkinson’s disease. Brain Research 1780, pages 147798.
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Daria Messelodi, Salvatore Nicola Bertuccio, Valentina Indio, Silvia Strocchi, Alberto Taddia, Salvatore Serravalle, Jessica Bandini, Annalisa Astolfi & Andrea Pession. (2021) iPSC-Derived Gaucher Macrophages Display Growth Impairment and Activation of Inflammation-Related Cell Death. Cells 10:11, pages 2822.
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Azucena Perez-Canamas, Hideyuki Takahashi, Jane A Lindborg & Stephen M Strittmatter. (2021) Fronto-temporal dementia risk gene TMEM106B has opposing effects in different lysosomal storage disorders . Brain Communications 3:1.
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Ayelet Vardi, Shifra Ben-Dor, Soo Min Cho, Ulrich Kalinke, Julia Spanier & Anthony H. Futerman. (2020) Mice defective in interferon signaling help distinguish between primary and secondary pathological pathways in a mouse model of neuronal forms of Gaucher disease. Journal of Neuroinflammation 17:1.
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Giulia Massaro, Michael P Hughes, Sammie M Whaler, Kerri-Lee Wallom, David A Priestman, Frances M Platt, Simon N Waddington & Ahad A Rahim. (2020) Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes. Human Molecular Genetics 29:12, pages 1933-1949.
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Ana R. P. Varela, Ana E. Ventura, Ana C. Carreira, Aleksander Fedorov, Anthony H. Futerman, Manuel Prieto & Liana C. Silva. (2017) Pathological levels of glucosylceramide change the biophysical properties of artificial and cell membranes. Physical Chemistry Chemical Physics 19:1, pages 340-346.
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Ayelet Vardi, Hila Zigdon, Anna Meshcheriakova, Andrés D Klein, Chen Yaacobi, Raya Eilam, Brandon M Kenwood, Ahad A Rahim, Giulia Massaro, Alfred H MerrillJrJr, Einat B Vitner & Anthony H Futerman. (2016) Delineating pathological pathways in a chemically induced mouse model of Gaucher disease. The Journal of Pathology 239:4, pages 496-509.
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John Marshall, Ying Sun, Dinesh S Bangari, Eva Budman, Hyejung Park, Jennifer B Nietupski, Amy Allaire, Mary A Cromwell, Bing Wang, Gregory A Grabowski, John P Leonard & Seng H Cheng. (2016) CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease. Molecular Therapy 24:6, pages 1019-1029.
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Melinda Barkhuizen, David G. Anderson & Anne F. Grobler. (2016) Advances in GBA-associated Parkinson's disease – Pathology, presentation and therapies. Neurochemistry International 93, pages 6-25.
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Elena M. Sánchez-Fernández, José M. García Fernández & Carmen Ortiz Mellet. (2016) Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, G M1 -gangliosidosis and Fabry diseases . Chemical Communications 52:32, pages 5497-5515.
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Arnold E. Stütz & Tanja M. Wrodnigg. 2016. 225 302 .
Marie T. Vanier, Catherine Caillaud & Thierry Levade. 2016. Inborn Metabolic Diseases. Inborn Metabolic Diseases 551 575 .

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