Antisense oligonucleotide therapy

Article | 01 July 2020

Suppression of proteolipid protein rescues Pelizaeus–Merzbacher disease

In a mouse model of the leukodystrophy Pelizaeus–Merzbacher disease, myelination, motor performance, respiratory function and lifespan are improved by suppressing proteolipid protein expression, suggesting PLP1 as a therapeutic target for human patients with this disease and, more broadly, antisense oligonucleotides as a pharmaceutical modality for treatment of myelin disorders.

Matthew S. Elitt
, Lilianne Barbar
& Paul J. Tesar

News & Views | 01 August 2012

Kill the messenger where it lives

A mutant repeating DNA sequence produces a toxic RNA molecule that causes the neuromuscular disorder myotonic dystrophy type 1. An ‘antisense’ therapy that targets this RNA in cell nuclei shows promise in mice. See Letter p.111

Peter K. Todd
& Henry L. Paulson

Letter | 01 August 2012

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

Nuclear-retained transcripts containing expanded repeats are shown to be sensitive to antisense silencing, and in a transgenic mouse model of myotonic dystrophy type 1, systemic administration of ASOs causes a rapid knockdown of the toxic RNA in skeletal muscle, correcting some hallmark features of the disease.