Targeting Huntingtin Expression in Patients with Huntington's Disease.

Adult Dose-Response Relationship, Drug Female Humans Huntingtin Protein / antagonists & inhibitors Huntington Disease / drug therapy Injections, Spinal Male Middle Aged Mutation Nucleotides / chemical synthesis Oligonucleotides / cerebrospinal fluid

Journal

The New England journal of medicine
ISSN: 1533-4406
Titre abrégé: N Engl J Med
Pays: United States
ID NLM: 0255562

Informations de publication

Date de publication:
13 06 2019
Historique:
pubmed: 7 5 2019
medline: 2 7 2019
entrez: 7 5 2019
Statut: ppublish

Résumé

Huntington's disease is an autosomal-dominant neurodegenerative disease caused by CAG trinucleotide repeat expansion in We conducted a randomized, double-blind, multiple-ascending-dose, phase 1-2a trial involving adults with early Huntington's disease. Patients were randomly assigned in a 3:1 ratio to receive HTT Of the 46 patients who were enrolled in the trial, 34 were randomly assigned to receive HTT Intrathecal administration of HTT

Sections du résumé

BACKGROUND
Huntington's disease is an autosomal-dominant neurodegenerative disease caused by CAG trinucleotide repeat expansion in
METHODS
We conducted a randomized, double-blind, multiple-ascending-dose, phase 1-2a trial involving adults with early Huntington's disease. Patients were randomly assigned in a 3:1 ratio to receive HTT
RESULTS
Of the 46 patients who were enrolled in the trial, 34 were randomly assigned to receive HTT
CONCLUSIONS
Intrathecal administration of HTT

Identifiants

DOI: 10.1056/NEJMoa1900907 PMID: 31059641
pubmed: 31059641
doi: 10.1056/NEJMoa1900907
doi:

Substances chimiques

HTT protein, human 0
HTT(RX) 0
Huntingtin Protein 0
Nucleotides 0
Oligonucleotides 0

Banques de données

ClinicalTrials.gov
['NCT02519036']

Types de publication

Clinical Trial, Phase I Clinical Trial, Phase II Journal Article Multicenter Study Randomized Controlled Trial Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

2307-2316

Subventions

Organisme : Medical Research Council
ID : MR/M008592/1
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/P008593/1
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/L010305/1
Pays : United Kingdom
Organisme : Medical Research Council
ID : G0801418
Pays : United Kingdom
Organisme : Wellcome Trust
ID : 200181/Z/15/Z
Pays : United Kingdom
Organisme : Medical Research Council
ID : G9810900
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/P001629/1
Pays : United Kingdom

Investigateurs

Peter McColgan (P)
Davina Hensman (D)
Rhia Ghosh (R)
Michael Flower (M)
Vincenzo Libri (V)
Edwina Saunders (E)
Lynn Raymond (L)
Joji Decolongon (J)
Tuan Li (T)
Panteha Fathinia (P)
Christina Lang (C)
Jan Lewerenz (J)
Katrin Lindenberg (K)
Albert C Ludolph (AC)
Ariane Schneider (A)
Sonja Trautmann (S)
Stefanie Uhl (S)
Patrick Weydt (P)
Barbara Kaminski (B)
Daniela Kaminski (D)
Rainer Hoffmann (R)
Sarah M von Hein (SM)
Siegfried Muhlack (S)
Ralf Gold (R)
Lucy Collins (L)
Sarah Mason (S)
Kirsten Scott (K)
Tom Stoker (T)
Julia Greenland (J)
Katie Andresen (K)
Mohan Shanmugam (M)
Mowafak Abdelghani (M)
Tolga Turgut (T)
Siofra Peeren (S)
Olga Colaco (O)
Rebecca Owens (R)
Sujamole Subin (S)
Eike Jakob Spruth (EJ)
Janna Beckmann (J)
Henriette Krug (H)
Anika Langenfurth (A)
Diana Crossley (D)
Nasreen Akhtar (N)
Jennie Gavin (J)
Jenny De Souza (J)
Thomas Massey (T)
Duncan McLauchlan (D)
Rebecca Cousins (R)
Dmitri Shastin (D)
Kathryn Peall (K)
Harsh Bhatt (H)
Andrew Davison (A)
Joanne Bagshawe (J)
Belinda Gunning (B)
Khalid Hamandi (K)

Commentaires et corrections

Type : CommentIn
Type : CommentIn
Type : CommentIn
Type : CommentIn
Type : CommentIn
Type : ErratumIn

Informations de copyright

Copyright © 2019 Massachusetts Medical Society.

Auteurs

Sarah J Tabrizi (SJ)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Blair R Leavitt (BR)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

G Bernhard Landwehrmeyer (GB)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Edward J Wild (EJ)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Carsten Saft (C)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Roger A Barker (RA)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Nick F Blair (NF)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

David Craufurd (D)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Josef Priller (J)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Hugh Rickards (H)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Anne Rosser (A)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Holly B Kordasiewicz (HB)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Christian Czech (C)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Eric E Swayze (EE)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Daniel A Norris (DA)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Tiffany Baumann (T)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Irene Gerlach (I)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Scott A Schobel (SA)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Erika Paz (E)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Anne V Smith (AV)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

C Frank Bennett (CF)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

Roger M Lane (RM)

From University College London (UCL) Huntington's Disease Centre, Department of Neurodegenerative Disease, Queen Square Institute of Neurology, UCL, and the U.K. Dementia Research Institute at UCL, London (S.J.T., E.J.W.), the Department of Clinical Neuroscience, Addenbrooke's Hospital, University of Cambridge, Cambridge (R.A.B., N.F.B.), Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, and the Division of Evolution and Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine, and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester (D.C.), the University of Edinburgh and the U.K. Dementia Research Institute, Edinburgh (J.P.), the Institute of Clinical Sciences, College of Medical and Dental Sciences, University Hospital Birmingham, Birmingham (H.R.), and the Cardiff University Brain Repair Group, Brain Repair and Intracranial Neurotherapeutics Unit, Neuroscience and Mental Health Research Institute and School of Biosciences, Cardiff (A.R.) - all in the United Kingdom; the Centre for Huntington's Disease, Department of Medical Genetics, and the Division of Neurology, Department of Medicine, University of British Columbia, and the Centre for Molecular Medicine and Therapeutics, B.C. Children's Hospital, Vancouver, Canada (B.R.L.); the Department of Neurology, Ulm University, Huntington's Disease Centre, Ulm (G.B.L.), the Department of Neurology, Huntington Center North Rhine-Westphalia, Ruhr University Bochum, St. Josef-Hospital, Bochum (C.S.), and the Department of Neuropsychiatry, Charité-Universitätsmedizin Berlin, Deutsches Zentrum für Neurodegenerative Erkrankungen, Berlin (J.P.) - all in Germany; Ionis Pharmaceuticals, Carlsbad, CA (H.B.K., E.E.S., D.A.N., T.B., E.P., A.V.S., C.F.B., R.M.L.); and F. Hoffmann-La Roche, Basel, Switzerland (C.C., I.G., S.A.S.).

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Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Targeting Huntingtin Expression in Patients...
questionsmedicales.fr › Phénomènes physiologiques › Phénomènes pharmacologiques et toxicologiques › Phénomènes toxicologiques › Relation dose-effet des médicaments › Targeting Huntingtin Expression in Patients...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Targeting Huntingtin Expression in Patients...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéine huntingtine › Targeting Huntingtin Expression in Patients...
questionsmedicales.fr › Troubles mentaux › Troubles neurocognitifs › Démence › Maladie de Huntington › Targeting Huntingtin Expression in Patients...
questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Voies d'administration de substances chimiques et des médicaments › Injections › Injections rachidiennes › Targeting Huntingtin Expression in Patients...
questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Adulte d'âge moyen › Targeting Huntingtin Expression in Patients...
questionsmedicales.fr › Phénomènes génétiques › Variation génétique › Mutation › Targeting Huntingtin Expression in Patients...
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Nucléotides › Targeting Huntingtin Expression in Patients...
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Nucléotides › Polynucléotides › Oligonucléotides › Targeting Huntingtin Expression in Patients...