CD33 splicing modulator 1

CD33 splicing modulator 1 is a small-molecule CD33 pre-mRNA splicing modulator with EC50 of 7.8 uM, enhances the exclusion of CD33 exon 2, reduces full-length V-domain containing CD33 protein.CD33/Siglec 3 is a myeloid lineage cell surface receptor that is known to regulate microglia activity.CD33 splicing modulator 1 reduces full-length V-domain containing CD33 protein in meyloid lineage cells (EC50=2.1 uM), increases exon 2 skipping in cellular mRNA pools.

1. Thomas A Chappie, et al. ACS Med Chem Lett. 2022 Jan 4;13(1):55-62.