Rett syndrome (RTT) is a severe neurological disorder caused by mutations in the gene
encoding methyl-CpG-binding protein 2 (MeCP2). Almost two decades of research into RTT …

Mutations in methyl CpG binding protein 2 (MeCP2) are the major cause of Rett syndrome
(RTT), a rare neurodevelopmental disorder with a notable period of developmental …

Although more than 98% of the human genome is non-coding, nearly all of the drugs on the
market target one of about 700 disease-related proteins. The historical reluctance to invest …

The long non-coding RNA (lncRNA) XIST establishes X chromosome inactivation (XCI) in
female cells in early development and thereafter is thought to be largely dispensable. Here …

Rett syndrome (RTT) is an X-linked neurodevelopmental disorder caused by loss-of-function
heterozygous mutations of methyl CpG-binding protein 2 (MECP2) on the X chromosome in …

RNA therapeutics (RNATx) aim to treat diseases, including cancer, by targeting or
employing RNA molecules for therapeutic purposes. Amongst the most promising targets …

The development of innovative methodologies to identify RNA binders has attracted
enormous attention in chemical biology and drug discovery. Although antibiotics targeting …

X inactivation (XCI) is triggered by upregulation of XIST, which coats the chromosome in cis,
promoting formation of a heterochromatic domain (**). XIST role beyond initiation of XCI is …

In female eutherian mammals, dosage compensation of X-linked gene expression is
achieved during development through transcriptional silencing of one of the two X …

Rare diseases are very difficult to study mechanistically and to develop therapies for
because of the scarcity of patients. Here, the rare neuro-metabolic disorder Rett syndrome …