High Concentration or Combined Treatment of Antisense Oligonucleotides for Spinal Muscular Atrophy Perturbed SMN2 Splicing in Patient Fibroblasts

High Concentration or Combined Treatment of Antisense Oligonucleotides for Spinal Muscular Atrophy Perturbed SMN2 Splicing in Patient Fibroblasts

Spinal muscular atrophy (SMA) is caused by survival motor neuron 1 SMN1 deletion. The survival motor neuron 2 (SMN2) encodes the same protein as SMN1 does, but it has a splicing defect of exon 7. Some antisense oligonucleotides (ASOs) have been proven to correct this defect. One of these, nusinersen...

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Bibliographic Details
Main Authors: Wijaya, Yogik Onky Silvana, Niba, Emma Tabe Eko, Nishio, Hisahide, Okamoto, Kentaro, Awano, Hiroyuki, Saito, Toshio, Takeshima, Yasuhiro, Shinohara, Masakazu
Format: Article
Language:English
Published: MDPI 2022
Subjects:
Medical Biochemistry and Metabolomics
Online Access:https://repository.ugm.ac.id/279049/1/High-Concentration-or-Combined-Treatment-of-Antisense-Oligonucleotides-for-Spinal-Muscular-Atrophy-Perturbed-SMN2-Splicing-in-Patient-FibroblastsGenes.pdf

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