Mesenchymal stromal cells (MSCs), also known as mesenchymal stem cells, have been
intensely investigated for clinical applications within the last decades. However, the majority …

A series of spectacular scientific discoveries and technological advances in the second half
of the 20th century have provided the basis for the ongoing genome editing revolution. The …

The versatility of RNA in sensing and interacting with small molecules, proteins and other
nucleic acids while encoding genetic instructions for protein translation makes it a powerful …

RNA base editing refers to the rewriting of genetic information within an intact RNA molecule
and serves various functions, such as evasion of the endogenous immune system and …

Summary Nonsense mutations, accounting for> 20% of disease-associated mutations, lead
to premature translation termination. Replacing uridine with pseudouridine in stop codons …

Clustered regularly interspaced short palindromic repeats (CRISPR)-based genome editors
are powerful tools in biology and hold great promise for the treatment of human diseases …

Members of the ADAR family of double-stranded RNA–binding proteins regulate one of the
most abundant RNA modifications in humans, the deamination of adenosine to inosine …

The exploitation of CRISPR-Cas systems especially CRISPR-Cas9 has led to radical
advances in genome editing, gene activation, gene repression, protein imaging, and …

Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas13 has drawn
broad interest to control gene expression and cell fate at the RNA level in general. Apart …

The past decade has yielded much success in the identification of risk genes for Autism
Spectrum Disorder (ASD), with many studies implicating loss-of-function (LoF) mutations …