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Mechanistically, mutations in GNAS1 lead to constitutive activation of GNAS1 and the coupled protein adenylyl cyclase, resulting in an increase in intracellular cyclic adenosine monophosphate (cAMP) levels [38, 39]

Weir DG, Scott JM

However, their therapeutic index is often narrowas exemplified by deferoxamine, which, despite being well-studied, is currently limited to treating acute iron poisoning and conditions involving chronic blood transfusions (e.g., thalassemia)

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Novel treatment of chronic graft-versus-host disease in mice using the ER stress reducer 4-phenylbutyric acid