A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference-Reference-Cited by-同舟云学术

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A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference

Published:2009-06 Issue:2 Volume:217 Page:312-319
ISSN:0014-4886
Container-title:Experimental Neurology
language:en
Short-container-title:Experimental Neurology

Author:

Lombardi Maria Stella,Jaspers Leonie,Spronkmans Christine,Gellera Cinzia,Taroni Franco,Di Maria Emilio,Donato Stefano Di,Kaemmerer William F.

Publisher

Elsevier BV

Subject

Developmental Neuroscience,Neurology

Reference30 articles.

1. Target selectivity in mRNA silencing;Aronin;Gene Ther.,2006

2. Huntington's Disease,2002

3. Rescue of polyglutamine-mediated cytotoxicity by double-stranded RNA-mediated RNA interference;Caplen;Hum. Mol. Genet.,2002

4. Normal huntingtin function: an alternative approach to Huntington's disease;Cattaneo;Nat. Rev. Neurosci.,2005

5. Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits;DiFiglia;Proc. Natl. Acad. Sci. U. S. A.,2007

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1. Huntingtin lowering therapeutics;Huntington's Disease;2024

2. DNA double-strand break-free CRISPR interference delays Huntington’s disease progression in mice;Communications Biology;2023-04-28

3. Allele-specific silencing as therapy for familial amyotrophic lateral sclerosis caused by the p.G376D TARDBP mutation;Brain Communications;2022-11-02

4. Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models;Nature Communications;2022-10-03

5. Development of versatile allele-specific siRNAs able to silence all the dominant dynamin 2 mutations;Molecular Therapy - Nucleic Acids;2022-09

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