Levy Gilad, Barak Boaz
The Sagol School of Neuroscience, Tel Aviv University, Tel Aviv, Israel.
The Sagol School of Neuroscience; The School of Psychological Sciences, Faculty of Social Sciences, Tel Aviv University, Tel Aviv, Israel.
Neural Regen Res. 2021 Mar;16(3):414-422. doi: 10.4103/1673-5374.293133.
Genetic neurodevelopmental disorders are characterized by abnormal neurophysiological and behavioral phenotypes, affecting individuals worldwide. While the subject has been heavily researched, current treatment options relate mostly to alleviating symptoms, rather than targeting the altered genome itself. In this review, we address the neurogenetic basis of neurodevelopmental disorders, genetic tools that are enabling precision research of these disorders in animal models, and postnatal gene-therapy approaches for neurodevelopmental disorders derived from preclinical studies in the laboratory.
遗传性神经发育障碍的特征是神经生理和行为表型异常，影响着世界各地的个体。尽管该主题已得到大量研究，但目前的治疗选择大多与缓解症状有关，而非针对改变的基因组本身。在本综述中，我们阐述了神经发育障碍的神经遗传学基础、有助于在动物模型中对这些疾病进行精准研究的遗传工具，以及源自实验室临床前研究的神经发育障碍的产后基因治疗方法。
