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Article

Transfer of genetic therapy across human populations: molecular targets for increasing patient coverage in repeat expansion diseases.

European journal of human genetics : EJHG - 1 Feb 2016

Varela Miguel A, Curtis Helen J, Douglas Andrew G L, Hammond Suzan M, O'Loughlin Aisling J, Sobrido Maria J, Scholefield Janine, Wood Matthew J A

Abstract excerpt

Allele-specific gene therapy aims to silence expression of mutant alleles through targeting of disease-linked single-nucleotide polymorphisms (SNPs). However, SNP linkage to disease varies between populations, making such molecular therapies applicable only to a subset of patients. Moreover, not all SNPs have the molecular features necessary for potent gene silencing. Here we provide knowledge to allow the...

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Share to Alleles
Share to DNA Repeat Expansion

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Genetic Diseases, Inborn

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Share to Genetics, Population

Share to Heterozygote

Share to Molecular Targeted Therapy

Source

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Identifiers

PMID: 25990798
DOI: 10.1038/ejhg.2015.94
PMCID: PMC4717213

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