Nusinersen |
| Catalog No.GC64293 |
Nusinersen은 SMN2 유전자의 pre-messenger RNA splicing을 수정하여 전체 길이의 SMN 단백질 생산을 증가시키는 안티센스 올리고뉴클레오티드 약물입니다.
Products are for research use only. Not for human use. We do not sell to patients.
Cas No.: 1258984-36-9
Sample solution is provided at 25 µL, 10mM.
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Nusinersen is an antisense oligonucleotide drug that modifies pre-messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein[1].
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].
[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.
Average Rating: 5 (Based on Reviews and 22 reference(s) in Google Scholar.)
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