| Description | Nusinersen, an antisense oligonucleotide drug, modifies the pre-messenger RNA (pre-mRNA) splicing of the SMN2 gene, thereby enhancing the production of the full-length Survival Motor Neuron (SMN) protein. |
| In vitro | Nusinersen is an SMN-ASO that increases SMN levels by blocking an intronic splice silencer in SMN2, thereby facilitating the exon 7 inclusion and generation of FL-SMN2 transcripts[2]. |
| molecular weight | N/A |
| CAS | 1258984-36-9 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |
| References | 1. Richard S Finkel, et al. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1723-1732. 2. Laura Torres-Benito, et al. NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice. Am J Hum Genet. 2019 Jul 3;105(1):221-230. |