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Medchemexpress LLC RNA, [2'-O-(2-methoxyethyl)](P-thio)(m5U-m5C-A-m5C-m5U-m5U-m5U-m5C-A-m5U-A-A-m5U-G-m5C-m5U-G-G) | 1258984-36-9 | 98.9% | 7127.3 | 1 MG
SDP

Supplier:  Medchemexpress LLC HY1129801MG

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Nusinersen sodium is an antisense oligonucleotide active molecule. It modifies the pre-messenger RNA splicing of the SMN2 gene, which promotes the production of full-length SMN protein. This action helps to improve spinal muscular atrophy. It is intended for research use only and not for sale to patients.

  • Antisense oligonucleotide active molecule.
  • Modifies pre-messenger RNA splicing of the SMN2 gene.
  • Promotes production of full-length SMN protein.
  • Improves spinal muscular atrophy.
  • Demonstrated improvement in motor function, preservation of αMN number, reduced motor neuron degeneration, and rescued skeletal muscle fiber size and oxidative metabolism in a severe SMA mouse model.

Catalog No. 50-004-39679


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