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Counter-balancing X-linked <i>Mecp2</i> hypofunction by hyperfunction ameliorates disease features in a model of Rett syndrome: implications for genetic therapies

2024-01-20

Abstract excerpt

<h4>ABSTRACT</h4> Treating monogenic neurodevelopmental disorders remains challenging and mostly symptomatic. X-linked disorders affecting women such as the postnatal neurodevelopmental disorder Rett syndrome (caused by mutations in the gene MECP2 ) have additional challenges due to dosage sensitivity and to cellular mosaicism caused by random X-chromosome inactivation. An approach to augment MECP2 expression f...

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Literature Corpus work
3f7524e8-0d58-5488-bc7b-39614d60b4f6
DOI
10.1101/2024.01.18.576265
Open publication

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Counter-balancing X-linked <i>Mecp2</i> hypofunction by hyperfunction ameliorates disease features in a model of Rett syndrome: implications for genetic therapiesDOI 10.1101/2024.01.18.576265
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