Prenatal and Postnatal Pharmacotherapy in Down Syndrome: The Search to Prevent or Ameliorate Neurodevelopmental and Neurodegenerative Disorders.

Prenatal and Postnatal Pharmacotherapy in Down Syndrome: The Search to Prevent or Ameliorate Neurodevelopmental and Neurodegenerative Disorders.
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唐氏综合征的产前和产后药物治疗:预防或改善神经发育和神经退行性疾病的研究。

DOI:
10.1146/annurev-pharmtox-041521-103641
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发表时间:
2022-01-06
影响因子:
12.5
通讯作者:
--
中科院分区:
医学1区
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--
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唐氏综合征(DS)-21号染色体三体-通常受到儿童和成人认知功能障碍和行为挑战以及老年人阿尔茨海默病的影响。没有经过证实的治疗方法专门针对这些认知或行为变化。然而,在啮齿动物模型和人类细胞模型的建立方面取得的进展有望支持此类治疗的发展。一个研究议程,强调识别过度表达的基因,有助于明显异常的认知和行为的模型系统构成了合理的下一步。这些基因表达的正常化可能会迎来一个成功治疗的时代,适用于DS患者的整个生命周期。
Those with Down syndrome (DS)—trisomy for chromosome 21—are routinely impacted by cognitive dysfunction and behavioral challenges in children and adults and Alzheimer’s disease in older adults. No proven treatments specifically address these cognitive or behavioral changes. However, advances in the establishment of rodent models and human cell models promise to support development of such treatments. A research agenda that emphasizes the identification of overexpressed genes that contribute demonstrably to abnormalities in cognition and behavior in model systems constitutes a rational next step. Normalizing expression of such genes may usher in an era of successful treatments applicable across the life span for those with DS.
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