Huntingtin Lowering Strategies for Disease Modification in Huntington's Disease.

Tabrizi, Sarah J; Ghosh, Rhia; Leavitt, Blair R

Tabrizi, Sarah J; Ghosh, Rhia; Leavitt, Blair R.

Afiliação

Tabrizi SJ; Huntington's Disease Centre, Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, University College London, London, UK; UK Dementia Research Institute (DRI) at UCL, London, UK. Electronic address: s.tabrizi@ucl.ac.uk.
Ghosh R; Huntington's Disease Centre, Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, University College London, London, UK.
Leavitt BR; UBC Centre for Huntington's Disease, Department of Medical Genetics and Centre for Molecular Medicine and Therapeutics, BC Children's Hospital, University of British Columbia, Vancouver, BC, Canada.

Neuron ; 101(5): 801-819, 2019 03 06.

Article em En | MEDLINE | ID: mdl-30844400

Assuntos

Terapia Genética/métodos; Proteína Huntingtina/genética; Doença de Huntington/terapia; Animais; Sistemas CRISPR-Cas; Humanos; Proteína Huntingtina/metabolismo; Doença de Huntington/etiologia

Palavras-chave

CRISPR/Cas9; HD biomarkers; Huntington's disease; RNAi; antisense oligonucleotides; drug delivery; genome editing; proteolysis-targeting chimeras; small-molecule splicing modulators; transcription activator-like effector nucleases; zinc-finger proteins

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Doença de Huntington / Proteína Huntingtina Idioma: En Ano de publicação: 2019 Tipo de documento: Article

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