ARTICLE | Distillery Therapeutics
Therapeutics: β-catenin (CTNNB1); wingless-type MMTV integration site family member 3A (WNT3A); 7-dehydrocholesterol reductase (DHCR7); glycogen synthase kinase
May 5, 2016 7:00 AM UTC
In vitro studies suggest activating Wnt/CTNNB1 signaling could help treat microcephaly associated with Smith-Lemli-Opitz syndrome, a developmental disorder caused by DHCR7 mutations. In patient-derive...