Supporting Rare Disease Research with the 2020 BeHEARD Awards
Type
Blog Post
...Deficiency (CTD) is a rare X-linked inherited neurometabolic condition and one of three Cerebral Creatine...affected signaling pathways and how they lead to neurological phenotypes observed.”
Using genetically-encoded...genetically-encoded biosensors and iPSC-derived cortical neurons from patients with GNB1 mutations, the lab hopes to...