Many diseases are caused by insufficient expression of mutated genes and would benefit
from increased expression of the corresponding protein. However, in drug development, it …

Oligonucleotide-based therapeutics have attracted attention as an emerging modality that
includes the modulation of genes and their binding proteins related to diseases, allowing us …

Herein, we report the systematic investigation of stereopure phosphorothioate (PS) and
phosphoryl guanidine (PN) linkages on siRNA-mediated silencing. The incorporation of …

A large hexanucleotide (G 4 C 2) repeat expansion in the first intronic region of C9orf72 is
the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal …

Duchenne muscular dystrophy (DMD) is a debilitating and fatal genetic disease affecting
1/5000 boys globally, characterized by progressive muscle breakdown and eventual death …

In the last decade, the field of oligonucleotide therapeutics has matured, with the regulatory
approval of several single-stranded and double-stranded RNA drugs. In this Perspective, I …

The growing market for therapeutic peptides and oligonucleotides (TIDES) draws attention
towards their manufacture, aiming at efficient and sustainable productive processes in view …

Synthetic antisense oligonucleotides (ASOs) and duplex RNAs (dsRNAs) are an
increasingly successful strategy for drug development. After a slow start, the pace of success …

Lipophilic oligonucleotide conjugates represent a powerful tool for nucleic acid cellular
delivery, and many methods for their synthesis have been developed over the past few …

Introduction lncRNAs are major players in regulatory networks orchestrating multiple cellular
functions, such as 3D chromosomal interactions, epigenetic modifications, gene expression …