Newly Funded FARA Grants, July 2019
FARA's most recent grant round was of very high quality, and included some very innovative research. After much work by the applicants, reviewers and Scientific Review Committee, the following grants will be funded:
Benoit D’Autreaux, Paris-Saclay University: “Cell-free high throughput screening assays for the discovery of compounds replacing frataxin in FA”
Hélène Puccio, the Institut de Génétique et de Biologie Moléculaire et Cellulaire: “Characterization of new humanized mouse model (TG(FXN)YG8Pook/800J) carrying 650-800 GAA repeats”
Mirella Dottori, University of Wollongong: “Nanoparticle-mediated gene delivery of frataxin to neurons”
Massimo Pandolfo, Université Libre de Bruxelles (ULB), Hôpital Erasme:“Induced pluripotent stem cells-derived neurons for translational research in Friedreich ataxia"
Thursday, September 5, 2019
Muscular Dystrophy Association Awards 25 Grants Totaling More Than $6.6 Million for Neuromuscular Disease Research
NEW YORK, Sept. 5, 2019 /PRNewswire. NEWS PROVIDED BY: Muscular Dystrophy Association, Sep 05, 2019.
Critical funding provided by MDA will support studies to further understand disease mechanisms, optimize and build upon existing therapies, and advance drug target identification, especially toward gene-targeted therapies -- research that will have translational and clinical application across many neuromuscular diseases
Jordi Magrane, PhD
Weill Cornell Medical College
Research grant, $300,000
Cell and non-cell autonomous effects of frataxin deficiency in FA pathophysiology
Critical funding provided by MDA will support studies to further understand disease mechanisms, optimize and build upon existing therapies, and advance drug target identification, especially toward gene-targeted therapies -- research that will have translational and clinical application across many neuromuscular diseases
Jordi Magrane, PhD
Weill Cornell Medical College
Research grant, $300,000
Cell and non-cell autonomous effects of frataxin deficiency in FA pathophysiology
Friedreich ataxia- pathogenesis and implications for therapies
Martin B. Delatycki, Sanjay I. Bidichandani, Neurobiology of Disease, 2019, 104606, doi: 10.1016/j.nbd.2019.104606.
Therapies that have been proposed include pharmaceuticals that increase frataxin levels, protein and gene replacement therapies, antioxidants, iron chelators and modulators of inflammation. Whilst no therapies have yet been approved for Friedreich ataxia, there is much optimism that the advances in the understanding of the pathogenesis of this disorder since the discovery its genetic basis, will result in approved disease modifying therapies in the near future.
Therapies that have been proposed include pharmaceuticals that increase frataxin levels, protein and gene replacement therapies, antioxidants, iron chelators and modulators of inflammation. Whilst no therapies have yet been approved for Friedreich ataxia, there is much optimism that the advances in the understanding of the pathogenesis of this disorder since the discovery its genetic basis, will result in approved disease modifying therapies in the near future.
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