Behavioral Abnormalities, Cognitive Impairments, Synaptic Deficits, and Gene Replacement Therapy in a CRISPR Engineered Rat Model of 5p15.2 Deletion Associated With Cri du Chat Syndrome

Behavioral Abnormalities, Cognitive Impairments, Synaptic Deficits, and Gene Replacement Therapy in a CRISPR Engineered Rat Model of 5p15.2 Deletion Associated With Cri du Chat Syndrome

Abstract The Cri du Chat Syndrome (CdCS), a devastating genetic disorder caused by a deletion on chromosome 5p, faces challenges in finding effective treatments and accurate animal models. Using CRISPR‐Cas9, a novel CdCS rat model with a 2q22 deletion is developed, mirroring a common genetic alterat...

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Bibliographic Details
Main Authors: Jingjing Shen, Yan Wang, Yang Liu, Junying Lan, Shuang Long, Yingbo Li, Di Chen, Peng Yu, Jing Zhao, Yongjun Wang, Shali Wang, Feng Yang
Format: Article
Language:English
Published: Wiley 2025-04-01
Series:Advanced Science
Subjects:
5p15.2 deletion
Cri du Chat syndrome
Ctnnd2
gene therapy
rat model
Online Access:https://doi.org/10.1002/advs.202415224
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https://doi.org/10.1002/advs.202415224

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