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

Molecular Therapy Nucleic Acids

Volumn 5, Issue , 2016, Pages e380-

  Ferri, Lorenzo ^a,b   Covello, Giuseppina ^c   Caciotti, Anna ^b   Guerrini, Renzo ^a,b   Denti, Michela Alessandra ^c   Morrone, Amelia ^a,b  

^a UNIVERSITY OF FLORENCE   (Italy)

^b UNIVERSITY OF FLORENCE   (Italy)

^c UNIVERSITY OF TRENTO   (Italy)

Author keywords

antisense therapy; exon skipping; Fabry disease; minigene; U1

Indexed keywords

ALPHA GALACTOSIDASE; ANTISENSE NUCLEAR U SMALL NUCLEAR RNA; COMPLEMENTARY RNA; CYCLOPHILIN A; MESSENGER RNA; RNA DIRECTED DNA POLYMERASE; SMALL NUCLEAR RNA; UNCLASSIFIED DRUG; GENOMIC DNA; U1 SMALL NUCLEAR RNA;

ANIMAL CELL; ARTICLE; CONTROLLED STUDY; COS-1 CELL LINE; ENZYME ACTIVITY; EXON; EXON SKIPPING; FABRY DISEASE; GENE EXPRESSION; GENE MUTATION; GENETIC TRANSFECTION; GENETIC VARIABILITY; HUMAN; INTRON; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; REAL TIME POLYMERASE CHAIN REACTION; RNA SPLICING; WESTERN BLOTTING; AGMK CELL LINE; ANALYSIS; ANTISENSE THERAPY; ENZYME ASSAY; MALE; REPORTER GENE;

EID: 85015187198     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2016.88     Document Type: Article

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