Expert Review

Prospect of gene therapy for developmental and epileptic encephalopathy

  • Taoyun JI
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  • Department of Pediatrics, The First Hospital of Peking University, Beijing 100034, China

Received date: 2023-06-20

  Online published: 2023-09-05

Abstract

Developmental and epileptic encephalopathy (DEE) is a group of heterogeneous disorders characterized by early-onset epilepsy, abnormal electroencephalography and developmental retardation or regression. The etiology of DEE is complex, with high disability rate and fatality rate. With the development of next-generation sequencing technology, more and more genetic causes related to DEE have been discovered, which also deepens the acknowledgement on the pathogenesis of DEE. These researches provide a basis for exploring different treatment methods, especially gene therapy. It is expected that gene therapy will be carried out in the future to improve the prognosis of DEE.

Cite this article

Taoyun JI . Prospect of gene therapy for developmental and epileptic encephalopathy[J]. Journal of Clinical Pediatrics, 2023 , 41(9) : 650 -655 . DOI: 10.12372/jcp.2023.23e0540

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