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Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease.
Caron, Nicholas S; Southwell, Amber L; Brouwers, Cynthia C; Cengio, Louisa Dal; Xie, Yuanyun; Black, Hailey Findlay; Anderson, Lisa M; Ko, Seunghyun; Zhu, Xiang; van Deventer, Sander J; Evers, Melvin M; Konstantinova, Pavlina; Hayden, Michael R.
Affiliation
  • Caron NS; Centre for Molecular Medicine and Therapeutics, Vancouver, British Columbia, Canada.
  • Southwell AL; Department of Medical Genetics, University of British Columbia, Vancouver, British Columbia, Canada.
  • Brouwers CC; BC Children's Hospital Research Institute, Vancouver, British Columbia, Canada.
  • Cengio LD; Centre for Molecular Medicine and Therapeutics, Vancouver, British Columbia, Canada.
  • Xie Y; Department of Medical Genetics, University of British Columbia, Vancouver, British Columbia, Canada.
  • Black HF; BC Children's Hospital Research Institute, Vancouver, British Columbia, Canada.
  • Anderson LM; Burnett School of Biomedical Sciences, College of Medicine, University of Central Florida, Orlando, Florida, USA.
  • Ko S; Department of Research & Development, uniQure biopharma B.V., Amsterdam, the Netherlands.
  • Zhu X; Centre for Molecular Medicine and Therapeutics, Vancouver, British Columbia, Canada.
  • van Deventer SJ; Centre for Molecular Medicine and Therapeutics, Vancouver, British Columbia, Canada.
  • Evers MM; Burnett School of Biomedical Sciences, College of Medicine, University of Central Florida, Orlando, Florida, USA.
  • Konstantinova P; Centre for Molecular Medicine and Therapeutics, Vancouver, British Columbia, Canada.
  • Hayden MR; Department of Medical Genetics, University of British Columbia, Vancouver, British Columbia, Canada.
Nucleic Acids Res ; 48(1): 36-54, 2020 01 10.
Article in En | MEDLINE | ID: mdl-31745548
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: RNA, Messenger / Huntington Disease / Parvovirinae / MicroRNAs / Molecular Targeted Therapy / Huntingtin Protein Language: En Journal: Nucleic Acids Res Year: 2020 Document type: Article Affiliation country: Canada Country of publication: United kingdom
Fulltext
Add to My VHL
Print
XML
PubMed Links
Search on Google

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: RNA, Messenger / Huntington Disease / Parvovirinae / MicroRNAs / Molecular Targeted Therapy / Huntingtin Protein Language: En Journal: Nucleic Acids Res Year: 2020 Document type: Article Affiliation country: Canada Country of publication: United kingdom