High Concentration of an ISS-N1-Targeting Antisense Oligonucleotide Causes Massive Perturbation of the Transcriptome

High Concentration of an ISS-N1-Targeting Antisense Oligonucleotide Causes Massive Perturbation of the Transcriptome

Intronic splicing silencer N1 (ISS-N1) located within <i>Survival Motor Neuron 2</i> (<i>SMN2</i>) intron 7 is the target of a therapeutic antisense oligonucleotide (ASO), nusinersen (Spinraza), which is currently being used for the treatment of spinal muscular atrophy (SMA),...

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Bibliographic Details
Main Authors: Eric William Ottesen, Diou Luo, Natalia Nikolaevna Singh, Ravindra Narayan Singh
Format: Article
Language:English
Published: MDPI AG 2021-08-01
Series:International Journal of Molecular Sciences
Subjects:
spinal muscular atrophy
SMA
survival motor neuron
SMN
ISS-N1
antisense oligonucleotide
Online Access:https://www.mdpi.com/1422-0067/22/16/8378

Internet

https://www.mdpi.com/1422-0067/22/16/8378

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