Allele-specific silencing of mutant Huntington’s disease gene
Author:
Publisher
Wiley
Subject
Cellular and Molecular Neuroscience,Biochemistry
Link
http://onlinelibrary.wiley.com/wol1/doi/10.1111/j.1471-4159.2008.05734.x/fullpdf
Reference20 articles.
1. Allele-specific silencing of a pathogenic mutant acetylcholine receptor subunit by RNA interference;Abdelgany;Hum. Mol. Genet.,2003
2. Ancestral differences in the distribution of the delta 2642 glutamic acid polymorphism is associated with varying CAG repeat lengths on normal chromosomes: insights into the genetic evolution of Huntington disease;Almqvist;Hum. Mol. Genet.,1995
3. Structure and expression of the Huntington’s disease gene: evidence against simple inactivation due to an expanded CAG repeat;Ambrose;Somat. Cell Mol. Genet.,1994
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. USA,2007
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1. Subtyping monogenic disorders: Huntington disease;Handbook of Clinical Neurology;2023
2. Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models;Nature Communications;2022-10-03
3. Chemical engineering of therapeutic siRNAs for allele-specific gene silencingin vivoin CNS;2022-07-02
4. Haplotype-specific insertion-deletion variations for allele-specific targeting in Huntington's disease;Molecular Therapy - Methods & Clinical Development;2022-06
5. SUPT4H1-edited stem cell therapy rescues neuronal dysfunction in a mouse model for Huntington’s disease;npj Regenerative Medicine;2022-01-19
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