Pharmacology of a central nervous system delivered 2′-O-methoxyethyl–modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates
F Rigo, SJ Chun, DA Norris, G Hung, S Lee… - The Journal of …, 2014 - Elsevier
Spinal muscular atrophy (SMA) is a debilitating neuromuscular disease caused by the loss
of survival of motor neuron (SMN) protein. Previously, we demonstrated that ISIS 396443, an
antisense oligonucleotide (ASO) targeted to the SMN2 pre-mRNA, is a potent inducer of
SMN2 exon 7 inclusion and SMN protein expression, and improves function and survival of
mild and severe SMA mouse models. Here, we demonstrate that ISIS 396443 is the most
potent ASO in central nervous system (CNS) tissues of adult mice, compared with several …
of survival of motor neuron (SMN) protein. Previously, we demonstrated that ISIS 396443, an
antisense oligonucleotide (ASO) targeted to the SMN2 pre-mRNA, is a potent inducer of
SMN2 exon 7 inclusion and SMN protein expression, and improves function and survival of
mild and severe SMA mouse models. Here, we demonstrate that ISIS 396443 is the most
potent ASO in central nervous system (CNS) tissues of adult mice, compared with several …
