Therapeutic Antisense Targeting of Huntingtin-Reference-Cited by-同舟云学术

Therapeutic Antisense Targeting of Huntingtin

Published:2020-02-01 Issue:2 Volume:39 Page:154-158
ISSN:1044-5498
Container-title:DNA and Cell Biology
language:en
Short-container-title:DNA and Cell Biology

Author:

Smith Anne V.¹,Tabrizi Sarah J.²

Affiliation:

1. Ionis Pharmaceuticals, Carlsbad, California.

2. Huntington's Disease Centre, Department of Neurodegenerative Disease, University College London (UCL) Queen Square Institute of Neurology, and the UK Dementia Research Institute at UCL, London, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Cell Biology,Genetics,Molecular Biology,General Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/dna.2019.5188

Reference10 articles.

1. Huntington disease

2. Pharmacology of Antisense Drugs

3. RNA-Targeted Therapeutics

4. Selecting, Acquiring, and Using Small Molecule Libraries for High‐Throughput Screening

5. Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia

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3. Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities;Human Gene Therapy;2022-01-01

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