RNA therapeutics (eg siRNA, oligonucleotides, mRNA, etc.) show great potential for the
treatment of a myriad of diseases. However, to reach their site of action in the cytosol or …

Abstract AGO2 (Argonaute 2, EIF2C2) is the only member in AGO family with catalytic activity
and of extreme importance during small RNAs guided gene silencing processes. The …

Bioengineering of viral vectors for therapeutic gene delivery is a pivotal strategy to reduce
doses, facilitate manufacturing, and improve efficacy and patient safety. Here, we …

Anti-CRISPR proteins are powerful tools for CRISPR–Cas9 regulation; the ability to
precisely modulate their activity could facilitate spatiotemporally confined genome …

Its remarkable ease and efficiency make the CRISPR (clustered regularly interspaced short
palindromic repeats) DNA editing machinery highly attractive as a new tool for experimental …

Adeno-associated virus (AAV) forms the basis for several commercial gene therapy products
and for countless gene transfer vectors derived from natural or synthetic viral isolates that …

Liver fibrosis, a form of scarring, develops in chronic liver diseases when hepatocyte
regeneration cannot compensate for hepatocyte death. Initially, collagen produced by …

The rapid development of CRISPR–Cas technologies brought a personalized and targeted
treatment of genetic disorders into closer reach. To render CRISPR-based therapies precise …

MicroRNAs (miRNAs) play a pivotal role in the regulation of cellular gene expression via the
conserved RNA interference (RNAi) mechanism. Biogenesis of the unusual miR-451 does …

Astrocytes, the most abundant cells in the mammalian brain, perform key functions and are
involved in several neurodegenerative diseases. The human immunodeficiency virus (HIV) …