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

• Main Authors: Samar Z. Rizvi (Author), Wing Suen Chan (Author), Eleonora Maino (Author), Sydney Steiman (Author), Georgiana Forguson (Author), Maya Klepfish (Author), Ronald D. Cohn (Author), Evgueni A. Ivakine (Author)
• Format: Book
• Published: Elsevier, 2024-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...