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Tytuł :
Insulin-like growth factor 2 (IGF2) protects against Huntington's disease through the extracellular disposal of protein aggregates.
Autorzy :
García-Huerta P; Faculty of Medicine, Biomedical Neuroscience Institute, University of Chile, Santiago, Chile.; Center for Geroscience, Brain Health and Metabolism, Santiago, Chile.; Program of Cellular and Molecular Biology, Institute of Biomedical Sciences, Sector B, Second Floor, Faculty of Medicine, University of Chile, Independencia 1027, P.O. Box 70086, Santiago, Chile.
Troncoso-Escudero P; Faculty of Medicine, Biomedical Neuroscience Institute, University of Chile, Santiago, Chile.; Center for Geroscience, Brain Health and Metabolism, Santiago, Chile.; Program of Cellular and Molecular Biology, Institute of Biomedical Sciences, Sector B, Second Floor, Faculty of Medicine, University of Chile, Independencia 1027, P.O. Box 70086, Santiago, Chile.; Center for Integrative Biology, Faculty of Sciences, University Mayor, Santiago, Chile.
Wu D; Department of Biomedical Sciences of Cells and Systems, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
Thiruvalluvan A; Department of Biomedical Sciences of Cells and Systems, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
Cisternas-Olmedo M; Faculty of Medicine, Biomedical Neuroscience Institute, University of Chile, Santiago, Chile.; Center for Geroscience, Brain Health and Metabolism, Santiago, Chile.; Center for Integrative Biology, Faculty of Sciences, University Mayor, Santiago, Chile.
Henríquez DR; Center for Geroscience, Brain Health and Metabolism, Santiago, Chile.; Department of Cell Biology, Faculty of Sciences, University of Chile, Santiago, Chile.
Plate L; Department of Molecular Medicine, The Scripps Research Institute, La Jolla, CA, USA.
Chana-Cuevas P; Faculty of Medical Sciences, University of Santiago de Chile, Santiago, Chile.
Saquel C; Center for Geroscience, Brain Health and Metabolism, Santiago, Chile.; Center for Integrative Biology, Faculty of Sciences, University Mayor, Santiago, Chile.
Thielen P; Department of Immunology and Infectious Diseases, Harvard School of Public Health, Boston, MA, 02115, USA.
Longo KA; Proteostasis Therapeutics, Cambridge, MA, USA.
Geddes BJ; Proteostasis Therapeutics, Cambridge, MA, USA.
Lederkremer GZ; Sagol School of Neuroscience, Tel Aviv University, Tel Aviv, Israel.; George Wise Faculty of Life Sciences, School of Molecular Cell Biology and Biotechnology, Tel Aviv University, Tel Aviv, Israel.
Sharma N; Sagol School of Neuroscience, Tel Aviv University, Tel Aviv, Israel.; George Wise Faculty of Life Sciences, School of Molecular Cell Biology and Biotechnology, Tel Aviv University, Tel Aviv, Israel.
Shenkman M; Sagol School of Neuroscience, Tel Aviv University, Tel Aviv, Israel.; George Wise Faculty of Life Sciences, School of Molecular Cell Biology and Biotechnology, Tel Aviv University, Tel Aviv, Israel.
Naphade S; Buck Institute for Research on Aging, Novato, CA, 94945, USA.
Sardi SP; Rare and Neurological Diseases Therapeutic Area, Sanofi, 49 New York Avenue, Framingham, MA, 01701, USA.
Spichiger C; Faculty of Sciences, Institute of Biochemistry and Microbiology, University Austral of Chile, Valdivia, Chile.
Richter HG; Faculty of Medicine, Institute of Anatomy, Histology and Pathology, University Austral of Chile, Valdivia, Chile.
Court FA; Center for Geroscience, Brain Health and Metabolism, Santiago, Chile.; Center for Integrative Biology, Faculty of Sciences, University Mayor, Santiago, Chile.; Buck Institute for Research on Aging, Novato, CA, 94945, USA.
Tshilenge KT; Buck Institute for Research on Aging, Novato, CA, 94945, USA.
Ellerby LM; Buck Institute for Research on Aging, Novato, CA, 94945, USA.
Wiseman RL; Department of Molecular Medicine, The Scripps Research Institute, La Jolla, CA, USA.
Gonzalez-Billault C; Center for Geroscience, Brain Health and Metabolism, Santiago, Chile.; Department of Cell Biology, Faculty of Sciences, University of Chile, Santiago, Chile.; Buck Institute for Research on Aging, Novato, CA, 94945, USA.
Bergink S; Department of Biomedical Sciences of Cells and Systems, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
Vidal RL; Faculty of Medicine, Biomedical Neuroscience Institute, University of Chile, Santiago, Chile. .; Center for Geroscience, Brain Health and Metabolism, Santiago, Chile. .; Center for Integrative Biology, Faculty of Sciences, University Mayor, Santiago, Chile. .
Hetz C; Faculty of Medicine, Biomedical Neuroscience Institute, University of Chile, Santiago, Chile. .; Center for Geroscience, Brain Health and Metabolism, Santiago, Chile. .; Program of Cellular and Molecular Biology, Institute of Biomedical Sciences, Sector B, Second Floor, Faculty of Medicine, University of Chile, Independencia 1027, P.O. Box 70086, Santiago, Chile. .; Buck Institute for Research on Aging, Novato, CA, 94945, USA. .
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Źródło :
Acta neuropathologica [Acta Neuropathol] 2020 Nov; Vol. 140 (5), pp. 737-764. Date of Electronic Publication: 2020 Jul 08.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Czasopismo naukowe
Tytuł :
Investigational Assay for Haplotype Phasing of the Huntingtin Gene.
Autorzy :
Svrzikapa N; Wave Life Sciences Ltd., Cambridge, MA 02138, USA.; Department of Paediatrics, Medical Sciences Division, University of Oxford, Oxford OX3 9DU, UK.
Longo KA; Wave Life Sciences Ltd., Cambridge, MA 02138, USA.
Prasad N; HudsonAlpha Discovery, Discovery Life Sciences, Huntsville, AL 35806, USA.; Genomic Services Laboratory, HudsonAlpha Institute for Biotechnology, Huntsville, AL 35806, USA.
Boyanapalli R; Wave Life Sciences Ltd., Cambridge, MA 02138, USA.
Brown JM; Wave Life Sciences Ltd., Cambridge, MA 02138, USA.
Dorset D; HudsonAlpha Discovery, Discovery Life Sciences, Huntsville, AL 35806, USA.; Genomic Services Laboratory, HudsonAlpha Institute for Biotechnology, Huntsville, AL 35806, USA.
Yourstone S; Q Solutions | EA Genomics, LLC, Morrisville, NC 27560, USA.
Powers J; Q Solutions | EA Genomics, LLC, Morrisville, NC 27560, USA.
Levy SE; HudsonAlpha Discovery, Discovery Life Sciences, Huntsville, AL 35806, USA.; Genomic Services Laboratory, HudsonAlpha Institute for Biotechnology, Huntsville, AL 35806, USA.
Morris AJ; Wave Life Sciences Ltd., Cambridge, MA 02138, USA.
Vargeese C; Wave Life Sciences Ltd., Cambridge, MA 02138, USA.
Goyal J; Wave Life Sciences Ltd., Cambridge, MA 02138, USA.
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Źródło :
Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2020 Sep 11; Vol. 19, pp. 162-173. Date of Electronic Publication: 2020 Sep 11 (Print Publication: 2020).
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Genotyping single nucleotide polymorphisms for allele-selective therapy in Huntington disease.
Autorzy :
Claassen DO; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Corey-Bloom J; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Dorsey ER; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Edmondson M; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Kostyk SK; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
LeDoux MS; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Reilmann R; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Rosas HD; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Walker F; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Wheelock V; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Svrzikapa N; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Longo KA; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Goyal J; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Hung S; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
Panzara MA; Vanderbilt University Medical Center (D.O.C.), Nashville, TN; University of California San Diego (J.C.-B.), La Jolla; University of Rochester Medical Center (E.R.D.), NY; HD Reach (M.E.), Raleigh, NC; Ohio State University (S.K.K.), Columbus; University of Memphis and Veracity Neuroscience, LLC (M.S.L.), TN; George-Huntingon-Institute & Department of Clinical Radiology University of Muenster (R.R.), Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, University of Tuebingen, Germany; Havard Medical School (H.D.R.), Massachusetts General Hospital, Boston; Wake Forest University School of Medicine (F.W.), Winston Salem, NC; University of California Davis Health (V.W.), Sacramento, CA; Wave Life Sciences USA, Inc. (N.S., K.A.L., J.G., S.H., M.A.P.), Cambridge, MA; and Department of Paediatrics (N.S.), Medical Sciences Division, University of Oxford, UK.
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Źródło :
Neurology. Genetics [Neurol Genet] 2020 May 14; Vol. 6 (3), pp. e430. Date of Electronic Publication: 2020 May 14 (Print Publication: 2020).
Typ publikacji :
Journal Article
Czasopismo naukowe

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