Antisense-based RNA therapy of factor V deficiency: In vitro and ex vivo rescue of a F5 deep-intronic splicing mutation

Blood

Volumn 122, Issue 23, 2013, Pages 3825-3831

  Nuzzo, Francesca ^a   Radu, Claudia ^b   Baralle, Marco ^c   Spiezia, Luca ^b   Hackeng, Tilman M ^a   Simioni, Paolo ^b   Castoldi, Elisabetta ^a  

^a MAASTRICHT UNIVERSITY   (Netherlands)

^b UNIVERSITY OF PADUA MEDICAL SCHOOL   (Italy)

^c INTERNATIONAL CENTRE FOR GENETIC ENGINEERING AND BIOTECHNOLOGY   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 5; DNA POLYMERASE; GENOMIC DNA; MESSENGER RNA; MORPHOLINO OLIGONUCLEOTIDE; RNA; SMALL NUCLEAR RNA; COMPLEMENTARY RNA; U7 SMALL NUCLEAR RNA;

ANTISENSE THERAPY; ARTICLE; BLOOD CLOTTING FACTOR 5 DEFICIENCY; BRAIN HEMORRHAGE; CELL DIFFERENTIATION; CELL VIABILITY; CLINICAL ASSESSMENT; CONTROLLED STUDY; COS-1 CELL LINE; CYTOTOXICITY; DISEASE SEVERITY; EX VIVO STUDY; GENETIC TRANSFECTION; HEP-G2 CELL LINE; HOMOZYGOSITY; HUMAN; HUMAN CELL; IMMUNOFLUORESCENCE; IN VITRO STUDY; INTRON; MEGAKARYOCYTE; PRIORITY JOURNAL; RNA SPLICING; SINGLE DRUG DOSE; SPLICING DEFECT; STEM CELL; STOP CODON; ANIMAL; BLOOD; CHLOROCEBUS AETHIOPS; COS 1 CELL LINE; FACTOR V DEFICIENCY; GENE THERAPY; GENETICS; HEPG2 CELL LINE; HOMOZYGOTE; METABOLISM; MUTATION; PATHOLOGY;

ANIMALS; CERCOPITHECUS AETHIOPS; COS CELLS; FACTOR V; FACTOR V DEFICIENCY; GENETIC THERAPY; HEP G2 CELLS; HOMOZYGOTE; HUMANS; INTRONS; MEGAKARYOCYTES; MUTATION; RNA SPLICING; RNA, ANTISENSE; RNA, SMALL NUCLEAR; TRANSFECTION;

EID: 84892848187     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2013-04-499657     Document Type: Article

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