The first published description of therapeutic applications of antisense oligonucleotide
(ASO) technology occurred in the late 1970s and was followed by the founding of …

To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …

Antisense oligonucleotides (ASOs) were first discovered to influence RNA processing and
modulate protein expression over two decades ago; however, progress translating these …

Antisense oligonucleotides (ASOs) bind sequence specifically to the target RNA and
modulate protein expression through several different mechanisms. The ASO field is an …

The presence of cell and tissue barriers together with the low biomembrane permeability of
various therapeutics often hampers systemic drug distribution; thus, most of the available …

Oligonucleotides (oligos) have been under clinical development for approximately the past
30 years, beginning with antisense oligonucleotides (ASOs) and apatmers and followed …

Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber
integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing …

Duchenne muscular dystrophy is a fatal neuromuscular disorder affecting around one in
3,500–5,000 male births that is characterized by progressive muscular deterioration. It is …

The field of gene therapy is striving more than ever to define a path to the clinic and the
market. Twenty gene therapy products have already been approved and over two thousand …

Splice-switching oligonucleotides (SSOs) are short, synthetic, antisense, modified nucleic
acids that base-pair with a pre-mRNA and disrupt the normal splicing repertoire of the …