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• W2090804804 abstract "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 <i>SMN2</i> pre-mRNA, is a potent inducer of <i>SMN2</i> 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 other chemically modified ASOs. We evaluated methods of ISIS 396443 delivery to the CNS and characterized its pharmacokinetics and pharmacodynamics in rodents and nonhuman primates (NHPs). Intracerebroventricular bolus injection is a more efficient method of delivering ISIS 396443 to the CNS of rodents, compared with i.c.v. infusion. For both methods of delivery, the duration of ISIS 396443–mediated <i>SMN2</i> splicing correction is long lasting, with maximal effects still observed 6 months after treatment discontinuation. Administration of ISIS 396443 to the CNS of NHPs by a single intrathecal bolus injection results in widespread distribution throughout the spinal cord. Based upon these preclinical studies, we have advanced ISIS 396443 into clinical development." @default.
• W2090804804 created "2016-06-24" @default.
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• W2090804804 date "2014-04-30" @default.
• W2090804804 modified "2023-10-09" @default.
• W2090804804 title "Pharmacology of a Central Nervous System Delivered 2′-<i>O</i>-Methoxyethyl–Modified Survival of Motor Neuron Splicing Oligonucleotide in Mice and Nonhuman Primates" @default.
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• W2090804804 doi "https://doi.org/10.1124/jpet.113.212407" @default.
• W2090804804 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/4056267" @default.
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