Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …

Antisense oligonucleotides (ASOs) interact with target RNAs via hybridization to modulate
gene expression through different mechanisms. ASO therapeutics are chemically modified …

Oligonucleotide therapeutics are attracting attention as a new treatment modality for a range
of diseases that have been difficult to target using conventional approaches. Technical …

Oligonucleotide therapies are currently experiencing a resurgence driven by advances in
backbone chemistry and discoveries of novel therapeutic pathways that can be uniquely and …

Synthetic therapeutic oligonucleotides (STO) represent the third bonafide platform for drug
discovery in the pharmaceutical industry after small molecule and protein therapeutics. So …

Recent years have seen unprecedented activity in the development of RNA-silencing
oligonucleotide therapeutics for metabolic diseases. Improved oligonucleotide design and …

Interactions of chemically modified nucleic acid therapeutics with plasma proteins play an
important role in facilitating distribution from the injection site to peripheral tissues by …

We determined the effect of attaching palmitate, tocopherol or cholesterol to PS ASOs and
their effects on plasma protein binding and on enhancing ASO potency in the muscle of …

Oligonucleotide-based therapies are a promising approach for treating a wide range of hard-
to-treat diseases, particularly genetic and rare diseases. These therapies involve the use of …

The reduction of survival motor neuron (SMN) protein causes spinal muscular atrophy
(SMA), an autosomal recessive neuromuscular disease. Nusinersen is an antisense …