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Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients.
Germline mutations in TMEM127 confer susceptibility to pheochromocytoma.
Linking SNPs to CAG repeat length in Huntington's disease patients.
Polymorphism, Single Nucleotide
CRISPRseek: a bioconductor package to identify target-specific guide RNAs for CRISPR-Cas9 genome-editing systems.
Allele-Selective Suppression of Mutant Huntingtin in Primary Human Blood Cells.
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities.
Polymorphism Single Nucleotide