Article
A combination treatment based on drug repurposing demonstrates mutation-agnostic efficacy in pre-clinical retinopathy models.
Nature communications - 15 Jul 2024
Leinonen Henri, Zhang Jianye, Occelli Laurence M, Seemab Umair, Choi Elliot H, L P Marinho Luis Felipe, Querubin Janice, Kolesnikov Alexander V, Galinska Anna, Kordecka Katarzyna, Hoang Thanh, Lewandowski Dominik, Lee Timothy T, Einstein Elliott E, Einstein David E, Dong Zhiqian, Kiser Philip D, Blackshaw Seth, Kefalov Vladimir J, Tabaka Marcin, Foik Andrzej, Petersen-Jones Simon M, Palczewski Krzysztof
Abstract excerpt
Inherited retinopathies are devastating diseases that in most cases lack treatment options. Disease-modifying therapies that mitigate pathophysiology regardless of the underlying genetic lesion are desirable due to the diversity of mutations found in such diseases. We tested a systems pharmacology-based strategy that suppresses intracellular cAMP and Ca2+ activity via G protein-coupled receptor (GPCR) modulation...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.