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siRNA p4A
- Development stage
- Preclinical
- Lead developer
- UMass Chan Medical School
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal, Intracerebroventricular
01
Overview
siRNA p4A is an experimental, allele-specific small interfering RNA (siRNA) designed for the treatment of Huntington's disease (HD). Developed by researchers at the University of Massachusetts Medical School, it specifically targets single nucleotide polymorphisms (SNPs) in the huntingtin (HTT) mRNA to selectively silence the mutant allele while sparing the wild-type allele. The "p4A" designation indicates that the siRNA contains a mismatch at position 4 of the guide strand (adenine) relative to the target sequence, a design strategy used to maximize discrimination between the mutant and wild-type transcripts. By reducing the expression of the toxic mutant huntingtin protein, siRNA p4A aims to slow or prevent the neurodegeneration associated with HD.
- Other names
- siRNA-p4AsiRNA-p-4AsiRNA-p 4Aallele-specific siRNA p4A
02
Targets
HTT mRNA (HTT / Huntingtin gene)
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