Lysosomal storage diseases (LSDs) are a group of rare, life-threatening genetic disorders,
usually caused by a dysfunction in one of the many enzymes responsible for intralysosomal …

MicroRNAs (miRNAs) are emerging as potent regulators of many biological processes,
including cellular differentiation and disease. Recently, miRNA has been directly involved in …

RNA interference (RNAi) denotes sequence-specific mRNA degradation induced by long
double-stranded RNA (dsRNA). RNAi is an ancient eukaryotic defense mechanism against …

Double-stranded RNA (dsRNA) can enter different pathways in mammalian cells, including
sequence-specific RNA interference (RNAi), sequence-independent interferon (IFN) …

Canonical RNA interference (RNAi) is sequence-specific mRNA degradation guided by
small interfering RNAs (siRNAs) made by RNase III Dicer from long double-stranded RNA …

Inhibition of virus replication by means of RNA interference has been reported for several
important human pathogens, including human immunodeficiency virus type 1 (HIV-1). RNA …

Promoter expressed long-hairpin RNAs (lhRNAs) that can be processed into multiple small
interfering RNA (siRNAs) are being considered as effective agents for treating rapidly …

This report is the outcome of an EFSA procurement aiming at investigating and summarising
the state of knowledge on (I) the mode-of-action of dsRNA and miRNA pathways,(II) the …

The present invention relates generally to a method of modi fying gene expression and to
synthetic genes for modifying endogenous gene expression in a cell, tissue or organ of a …

RNA Interference (RNAi) effectors have been used to inhibit rogue RNAs in mammalian
cells. However, rapidly evolving sequences such as the human immunodeficiency virus type …