antisense oligonucleotide targeting rs72239206

This antisense oligonucleotide (ASO) is an experimental allele-specific therapeutic candidate designed for the treatment of Huntington's disease (HD). It selectively targets the mutant huntingtin (HTT) transcript by recognizing the rs72239206 insertion/deletion (indel) polymorphism, a genomic variant frequently found in cis with the pathogenic CAG repeat expansion. The ASO utilizes an RNase H-mediated mechanism to degrade the mutant HTT mRNA, thereby reducing the production of the neurotoxic mutant huntingtin protein. Crucially, this allele-specific approach spares the wild-type HTT mRNA, preserving the expression of the normal huntingtin protein which is essential for neuronal health and development. This selectivity addresses a major limitation of non-selective HTT-lowering strategies, which may risk adverse effects by suppressing the physiological functions of the wild-type protein.

Other names

rs72239206-targeted ASOrs-72239206-targeted ASOrs 72239206-targeted ASOHTT-rs72239206 ASOHTT-rs-72239206 ASOHTT-rs 72239206 ASOallele-specific HTT ASO