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

Intronic splicing silencer N1 (ISS-N1) located within Survival Motor Neuron 2 (SMN2) 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), a leading genetic disease associated...

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Detalles Bibliográficos
Autores principales: Ottesen, Eric William, Luo, Diou, Singh, Natalia Nikolaevna, Singh, Ravindra Narayan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8395096/
https://www.ncbi.nlm.nih.gov/pubmed/34445083
http://dx.doi.org/10.3390/ijms22168378

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8395096/
https://www.ncbi.nlm.nih.gov/pubmed/34445083
http://dx.doi.org/10.3390/ijms22168378

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