Preclinical models of Lennox-Gastaut syndrome: Progress and priorities

Lennox-Gastaut综合征的临床前模型:进展与重点

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Abstract

In the last decade, the increasing availability of genetic testing and rapid discovery of genes causing developmental and epileptic encephalopathies such as Lennox-Gastaut syndrome (LGS), together with technology such as CRISPR/Cas9 gene editing, has enabled the generation of new preclinical models, particularly using mice. Mice engineered to express disease-causing genetic changes, through either gene "knock-out" or "knock-in" of specific variants, recapitulate key features of LGS. In this review, we review past and recently developed preclinical models of LGS, and we highlight select insights already gleaned in preclinical studies. We argue that the availability of these new preclinical models should prompt renewed attention to basic and translational studies aimed at understanding fundamental biological mechanisms that enable secondary network evolution to LGS as well as novel, mechanistically informed therapeutic strategies.

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