中国神经再生研究(英文版) ›› 2021, Vol. 16 ›› Issue (3): 414-422.doi: 10.4103/1673-5374.293133

• 综述:神经损伤修复保护与再生 • 上一篇    下一篇

遗传性神经发育障碍的产后治疗方法

  

  • 出版日期:2021-03-15 发布日期:2020-12-16

Postnatal therapeutic approaches in genetic neurodevelopmental disorders

Gilad Levy1, Boaz Barak1, 2 , *   

  1. 1 The Sagol School of Neuroscience, Tel Aviv University, Tel Aviv, Israel;  2 The School of Psychological Sciences, Faculty of Social Sciences, Tel Aviv University, Tel Aviv, Israel
  • Online:2021-03-15 Published:2020-12-16
  • Contact: Boaz Barak, boazba@tauex.tau.ac.il.
  • Supported by:
    This work was supported by grants from Fritz Thyssen Stiftung, BrainBoost Innovation Center by Sagol School of Neuroscience at TAU, and SPARK Tel Aviv. GL was supported by the Eshkol Fellowship from The Ministry of Science and Technology, Israel. BB was the recipient of The Alon Fellowship for outstanding young researchers awarded by the Israeli Council for Higher Education.

摘要: https://orcid.org/0000-0001-9724-4578 (Boaz Barak)

Abstract: 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. 

Key words: adeno-associated virus, CRISPR-Cas9, developmental window, genetic therapy, mouse models, neurodevelopmental disorders, non-human primates, preclinical studies