Article

Juvenile reinstatement of TCF4 in Pitt-Hopkins syndrome model mice reveals a critical window for genetic intervention

2025-12-24

Abstract excerpt

<h4>ABSTRACT</h4> Pitt-Hopkins syndrome (PTHS) is a neurodevelopmental disorder caused by haploinsufficiency of TCF4 which encodes transcription factor 4. As PTHS therapeutics advance toward clinical trials, identifying the optimal timing for treatment is crucial. Our previous research demonstrated that restoring TCF4 during embryonic or neonatal stages, corresponding to prenatal or neonatal periods in humans, i...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 22fb91a4-94ff-5971-aa1a-09b6792028a3
DOI: 10.64898/2025.12.23.696277

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Juvenile reinstatement of TCF4 in Pitt-Hopkins syndrome model mice reveals a critical window for genetic interventionDOI 10.64898/2025.12.23.696277Read

Rescue of behavioral and electrophysiological phenotypes in a Pitt-Hopkins syndrome mouse model by genetic restoration of Tcf4 expressionSemantic proximity Read

Rescue of behavioral and electrophysiological phenotypes in a Pitt-Hopkins syndrome mouse model by genetic restoration of Tcf4 expression.Semantic proximity Read

Common Pathophysiology in Multiple Mouse Models of Pitt-Hopkins Syndrome.Semantic proximity Read

Enriched environment ameliorates adult hippocampal neurogenesis deficits in Tcf4 haploinsufficient miceSemantic proximity Read

<i>Tcf4</i> encodescortical differentiation during developmentSemantic proximity Read

<i>Tcf4</i> Deficiency Causes Recurrent Seizures in MiceSemantic proximity Read

MeCP2 gene therapy ameliorates disease phenotype in mouse model for Pitt Hopkins syndrome.Semantic proximity Read

Clemastine fumarate enhances myelination and promotes functional recovery in a syndromic ASD mouse model of Pitt-Hopkins SyndromeSemantic proximity Read

[Clinical and genetic characteristics of children with Pitt-Hopkins syndrome caused by TCF4 gene variants].Semantic proximity Read

Pitt-Hopkins Syndrome: A Review of Current Literature, Clinical Approach, and 23-Patient Case Series.Semantic proximity Read

TCF4 Mutations Disrupt Synaptic Function Through Dysregulation of RIMBP2 in Patient-Derived Cortical Neurons.Semantic proximity Read

Phenotype and natural history in 101 individuals with Pitt-Hopkins syndrome through an internet questionnaire system.Semantic proximity Read