BioCentury
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...