Multi-gene duplication removal in an engineered human cellular MECP2 duplication syndrome model with an IRAK1-MECP2 duplication

Recent progress in genome editing technologies has catalyzed the generation of sophisticated cell models; however, the precise modeling of copy-number variation (CNV) diseases remains a significant challenge despite their substantial prevalence in the human population. To overcome this barrier, we h...

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Prif Awduron:	Samar Z. Rizvi, Wing Suen Chan, Eleonora Maino, Sydney Steiman, Georgiana Forguson, Maya Klepfish, Ronald D. Cohn, Evgueni A. Ivakine
Fformat:	Artigo
Iaith:	Inglês
Cyhoeddwyd:	Elsevier 2024-12-01
Cyfres:	Molecular Therapy: Nucleic Acids
Pynciau:	MT: RNA/DNA Editing MECP2 duplication syndrome IRAK1 disease modeling HAP1 CRISPR-Cas9
Mynediad Ar-lein:	http://www.sciencedirect.com/science/article/pii/S2162253124002439
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