我们距离基于环磷酸腺苷（cAMP）和环磷酸鸟苷（cGMP）理论的脆性X综合征药物治疗还有多远？
How close are we to a cAMP- and cGMP-theory-based pharmacological therapy for fragile X syndrome?
作者信息
Bardoni Barbara, Gwizdek Carole, Maurin Thomas
机构信息
Inserm U1323CNRS UMR7275, Institut de Pharmacologie Moléculaire et Cellulaire, 06560 Valbonne, France.
Inserm U1323CNRS UMR7275, Institut de Pharmacologie Moléculaire et Cellulaire, 06560 Valbonne, France.
出版信息
Cell Rep Med. 2025 Feb 18;6(2):101972. doi: 10.1016/j.xcrm.2025.101972.
Abstract
Recent advances in targeting cAMP and cGMP pathways offer hope for treating fragile X syndrome, a leading cause of inherited intellectual disability. PDE4 and PDE2 inhibitors have shown promise in animal models, improving memory, social behavior, and cognitive function. Clinical trials are underway, raising optimism for future therapies.
摘要
靶向环磷酸腺苷（cAMP）和环磷酸鸟苷（cGMP）信号通路的最新进展为治疗脆性X综合征带来了希望，脆性X综合征是遗传性智力残疾的主要原因。磷酸二酯酶4（PDE4）和磷酸二酯酶2（PDE2）抑制剂在动物模型中已显示出前景，可改善记忆、社交行为和认知功能。临床试验正在进行中，这让人们对未来的治疗方法充满乐观。
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How close are we to a cAMP- and cGMP-theory-based pharmacological therapy for fragile X syndrome?我们距离基于环磷酸腺苷（cAMP）和环磷酸鸟苷（cGMP）理论的脆性X综合征药物治疗还有多远？
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