An engineered U1 small nuclear RNA rescues splicing-defective coagulation F7 gene expression in mice

Journal of Thrombosis and Haemostasis

Volumn 12, Issue 2, 2014, Pages 177-185

  Balestra, D ^a   Faella, A ^b   Margaritis, P ^b,c   Cavallari, N ^a   Pagani, F ^d   Bernardi, F ^a   Arruda, V R ^b,c   Pinotti, M ^a  

^a UNIVERSITY OF FERRARA   (Italy)

^b CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^c UNIVERSITY OF PENNSYLVANIA   (United States)

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

Author keywords

Factor VII deficiency; Genetic diseases; Mouse; Mutation; RNA splicing; U1 snRNA

Indexed keywords

ALANINE AMINOTRANSFERASE; BLOOD CLOTTING FACTOR 7; MESSENGER RNA PRECURSOR; PARVOVIRUS VECTOR; SMALL NUCLEAR RNA; SMALL NUCLEAR RNA U1; UNCLASSIFIED DRUG;

ALANINE AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BLOOD CLOTTING FACTOR 7 DEFICIENCY; CONTROLLED STUDY; DOSE RESPONSE; FOLLOW UP; GENE EXPRESSION; GENE THERAPY; IN VIVO STUDY; LIVER CELL; LIVER TOXICITY; LOW DRUG DOSE; MALE; MOUSE; MUTANT; MUTATION; NONHUMAN; NUCLEIC ACID ANALYSIS; NUCLEOTIDE SEQUENCE; PLASMID; PRIORITY JOURNAL; RNA SPLICING; SEROTYPE; SPLICING DEFECT; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; BASE SEQUENCE; BLOOD COAGULATION DISORDERS; DNA PRIMERS; GENE EXPRESSION; GENETIC VECTORS; MALE; MICE; MICE, INBRED C57BL; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA SPLICING; RNA, SMALL NUCLEAR;

EID: 84893549419     PISSN: 15387933     EISSN: 15387836     Source Type: Journal    
DOI: 10.1111/jth.12471     Document Type: Article

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