Pharmacological cAMP stimulation via prostaglandin receptors rescues ciliary defects in CEP290-deficient patient and mouse models
France de Malglaive, Iris Barny, Shahd Machroub, Lucas Fares‐Taie, Margarita Cano, Nicolas Goudin, Tania Attié‐Bitach, Josseline Kaplan, Isabelle Perrault, Luis Briseño-Roa, Jean‐Michel Rozet, Jean‐Philippe Annereau
This study shows that Taprenepag, a PGE2 receptor agonist, improves cilia formation and light sensitivity in CEP290-deficient models by stimulating cAMP synthesis.
bioRxiv · Oct 6, 2023 · PreprintGenetics