Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in
the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice …

Recent advances in the generation, purification and cellular delivery of RNA have enabled
development of RNA-based therapeutics for a broad array of applications. RNA therapeutics …

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …

Antisense oligonucleotides (ASOs) are used to selectively inhibit the translation of disease-
associated genes via Ribonuclease H (RNaseH)-mediated cleavage or steric hindrance …

RNA therapy refers to the treatment or prevention of diseases using RNA-based molecules.
The recent advent of a series of effective messenger RNA-based vaccines in response to the …

Spinal muscular atrophy (SMA) encompasses a group of autosomal recessively inherited
degenerative neuromuscular disorders. They range in severity from neonatal onset with …

Ribonucleic acid (RNA) therapeutics are being actively researched as a therapeutic
modality in preclinical and clinical studies. They have become one of the most ubiquitously …

The availability of disease modifying therapies for spinal muscular atrophy (SMA) has
created an urgent need to identify clinically meaningful biomarkers. Biomarkers present a …

A few protein kinases and phosphatases regulate tau protein phosphorylation and an
imbalance in their enzyme activity results in tau hyper-phosphorylation. Aberrant tau …

Genetic modification of living organisms has been a prosperous activity for research and
development of agricultural, industrial and biomedical applications. Three decades have …