CD33 splicing modulator 1 (Compound 1) is a CD33 splicing modulator. CD33/Siglec 3 is a myeloid lineage cell surface receptor that is known to regulate microglia activity. CD33 splicing modulator 1 increases exon 2 skipping in cellular mRNA pools. CD33 splicing modulator 1 has the potential for the research of neurodegenerative disease including Alzheimer's disease.
References:
[1]. Chappie TA, et al. Discovery of Small-Molecule CD33 Pre-mRNA Splicing Modulators. ACS Med Chem Lett. 2022;13(1):55-62.
















