Histone lysine methyltransferase-related neurodevelopmental disorders: current knowledge and saRNA future therapies

• Published in: Frontiers in cell and developmental biology Vol. 11; p. 1090046
• Main Authors: Roth, Charlotte, Kilpinen, Helena, Kurian, Manju A, Barral, Serena
• Format: Journal Article
• Language: English
• Published: Switzerland Frontiers Media S.A 27.02.2023
• Subjects: brain organoids; Cell and Developmental Biology; epigenetics (chromatin remodeling); histone lysine methyltransferases (HKMTs); neurodevelopmental disorders (NDDs); small-activating RNA (saRNA); epigenetics (chromatin remodeling); small-activating RNA (saRNA); histone lysine methyltransferases (HKMTs); brain organoids; neurodevelopmental disorders (NDDs)

Neurodevelopmental disorders encompass a group of debilitating diseases presenting with motor and cognitive dysfunction, with variable age of onset and disease severity. Advances in genetic diagnostic tools have facilitated the identification of several monogenic chromatin remodeling diseases that c...