Roadmap for developing biologically inspired therapeutics for genetic brain disorders.
Roadmap for developing biologically inspired therapeutics for genetic brain disorders.
复制标题
开发遗传性脑部疾病的生物启发疗法的路线图。
DOI：
10.1016/j.molmed.2023.07.005
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发表时间：
2023
影响因子：
13.6
通讯作者：
Paşca,SergiuP
中科院分区：
文献类型：
--
作者：
Imaizumi,Kent;Paşca,SergiuP
A requirement for developing effective therapeutics is to understand the biological events leading to disease. For neuropsychiatric disorders, this will involve understanding how genetic events perturb neural cells and cause circuit dysfunction, impacting behavior. It is now possible to derive human cellular and circuit models of the nervous system to study pathophysiology: organoids to model cell diversity; assembloids to capture cell–cell interactions; transplantation of organoids into behavior-capable circuits. A multilevel approach going from testing cells to probing and manipulating circuits in vivo is emerging as a new way to study disease and offers a glimpse into future therapeutic development.
