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Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations

Fabry disease is a rare X-linked lysosomal storage disorder caused by deficiency of the α-galactosidase A (α-Gal A) enzyme, which is encoded by the GLA gene. GLA transcription in humans produces a major mRNA encoding α-Gal A and a minor mRNA of unknown function, which retains a 57-nucleotide-long cr...

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Detalles Bibliográficos
Autores principales:	Ferri, Lorenzo, Covello, Giuseppina, Caciotti, Anna, Guerrini, Renzo, Denti, Michela Alessandra, Morrone, Amelia
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2016
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5095687/ https://www.ncbi.nlm.nih.gov/pubmed/27779620 http://dx.doi.org/10.1038/mtna.2016.88

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