Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry. - sprookjes - yvandenbroek - TriplyDB

Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry.

Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry.

http://www.wikidata.org/entity/Q39791825

about

Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing.Haplotype-based stratification of Huntington's disease.Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease.The targetable A1 Huntington disease haplotype has distinct Amerindian and European origins in Latin America.Huntington disease reduced penetrance alleles occur at high frequency in the general population.Study of the Huntington's disease IT-15 gene in different ethnic groups in Ecuador.Therapies targeting DNA and RNA in Huntington's disease.PURA, the gene encoding Pur-alpha, member of an ancient nucleic acid-binding protein family with mammalian neurological functions.Huntington’s Disease Clinical Trials Corner: February 2018 Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic.A human huntingtin SNP alters post-translational modification and pathogenic proteolysis of the protein causing Huntington disease.

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P2860

Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry.

http://www.wikidata.org/entity/Q39791825

description

2015 nî lūn-bûn

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Huntingtin Haplotypes Provide ...... Patients of European Ancestry.

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Huntingtin Haplotypes Provide ...... Patients of European Ancestry.

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Molecular Therapy

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Huntingtin Haplotypes Provide ...... Patients of European Ancestry

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Annamaria Griguoli

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Betty Nguyen

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Chris Kay

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Crystal N Doty

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Jennifer A Collins

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Michael R Hayden

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Simon C Warby

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P2860

An integrated map of genetic variation from 1,092 human genomes

A map of human genome variation from population-scale sequencing

CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup

A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes.

Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes

Increased apoptosis and early embryonic lethality in mice nullizygous for the Huntington's disease gene homologue

Inactivation of the mouse Huntington's disease gene homolog Hdh

Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients

Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease

Inactivation of Hdh in the brain and testis results in progressive neurodegeneration and sterility in mice

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1759-1771

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10.1038/MT.2015.128

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11

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23

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Ferdinando Squitieri

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280389419

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Huntington disease

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4817952

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