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Development of 2′-O-Methyl and LNA Antisense Oligonucleotides for SMN2 Splicing Correction in SMA Cells

Spinal muscular atrophy (SMA) is a devastating neurodegenerative disease caused by mutations in the SMN1 gene. Existing therapies demonstrate positive results on SMA patients but still might be ameliorated in efficacy and price. In the presented study we designed antisense oligonucleotides (AONs), t...

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Detalles Bibliográficos
Autores principales:	Maretina, Marianna, Il’ina, Arina, Egorova, Anna, Glotov, Andrey, Kiselev, Anton
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10669464/ https://www.ncbi.nlm.nih.gov/pubmed/38002071 http://dx.doi.org/10.3390/biomedicines11113071

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