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Proof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52

Marfan syndrome is one of the most common dominantly inherited connective tissue disorders, affecting 2–3 in 10,000 individuals, and is caused by one of over 2800 unique FBN1 mutations. Mutations in FBN1 result in reduced fibrillin-1 expression, or the production of two different fibrillin-1 monomer...

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Detalles Bibliográficos
Autores principales:	Cale, Jessica M., Greer, Kane, Fletcher, Sue, Wilton, Steve D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8037683/ https://www.ncbi.nlm.nih.gov/pubmed/33801742 http://dx.doi.org/10.3390/ijms22073479

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