Epigenetics in rare neurological diseases
CT Roberts, KS Arezoumand… - Frontiers in Cell and …, 2024 - frontiersin.org
Rare neurological diseases include a vast group of heterogenous syndromes with primary
impairment (s) in the peripheral and/or central nervous systems. Such rare disorders may …
impairment (s) in the peripheral and/or central nervous systems. Such rare disorders may …
Dose-dependent nuclear delivery and transcriptional repression with a cell-penetrant MeCP2
The vast majority of biologic-based therapeutics operate within serum, on the cell surface, or
within endocytic vesicles, in large part because proteins and nucleic acids fail to efficiently …
within endocytic vesicles, in large part because proteins and nucleic acids fail to efficiently …
MeCP2 is a naturally supercharged protein with cell membrane transduction capabilities
AV Beribisky, A Huber, V Sarne, A Spittler… - Protein …, 2024 - Wiley Online Library
The intrinsically disordered protein MeCP2 is a global transcriptional regulator encoded by
the MECP2 gene. Although the structured domains of MeCP2 have been the subject of …
the MECP2 gene. Although the structured domains of MeCP2 have been the subject of …
Generation and characterization of a human neuronal in vitro model for Rett Syndrome using a direct reprogramming method
A Huber, V Sarne, AV Beribisky… - Stem Cells and …, 2024 - liebertpub.com
Rett Syndrome (RTT) is a severe neurodevelopmental disorder, afflicting 1 in 10,000 female
births. It is caused by mutations in the X-linked methyl-CpG-binding protein gene (MECP2) …
births. It is caused by mutations in the X-linked methyl-CpG-binding protein gene (MECP2) …