Official websites use .gov
A
.gov website belongs to an official
government organization in the United States.
Secure .gov websites use HTTPS
A lock (
) or https:// means you've safely
connected to the .gov website. Share sensitive
information only on official, secure websites.
FHNA and S-cEt improve allele selectivity when incorporated at positions 5 and 6 of ASO A1. Dose-dependent reduction of muHTT and wtHTT mRNA in human GM04022 fibroblasts for (A) ASO A25 (FHNA at position 6) and A26 (S-cEt at position 5) (B) ASO A27 (FHNA at position 6) and A28 (S-cEt at position 6). Summary of sequence, activity and selectivity for ASOs (C) A1, A25, A26 and (D) A27 and A28. Underlined nucleotide indicates position across from the SNP site. Error bars are in ±SD.
