Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing

Nature Medicine

Volumn 9, Issue 8, 2003, Pages 1015-1019

  Chao, Hengjun ^a   Mansfield, S Gary ^b   Bartel, Robert C ^b   Hiriyanna, Suja ^b   Mitchell, Lloyd G ^b   Garcia Blanco, Mariano A ^b   Walsh, Christopher E ^a  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^b Intronn Inc   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; COMPLEMENTARY DNA; GENE PRODUCT; MESSENGER RNA; RECOMBINANT BLOOD CLOTTING FACTOR 8; RNA;

ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DNA REPAIR; F8 GENE; GENE; GENE THERAPY; GENE TRANSFER; GENETIC DISORDER; HEMOPHILIA A; HUMAN; HUMAN CELL; KNOCKOUT MOUSE; MOUSE; NONHUMAN; PHENOTYPE; PHENOTYPE CORRECTION; PRIORITY JOURNAL; RNA REPAIR; SPLICEOSOME; SPLICEOSOME MEDIATED RNA TRANS SPLICING; TRANS SPLICING;

ANIMALS; BLOOD COAGULATION; CELL LINE; DISEASE MODELS, ANIMAL; FACTOR VIII; GENE THERAPY; HEMOPHILIA A; HUMANS; MICE; MICE, KNOCKOUT; PHENOTYPE; RNA; RNA SPLICING; SPLICEOSOMES;

ANIMALIA;

EID: 0042388694     PISSN: 10788956     EISSN: None     Source Type: Journal    
DOI: 10.1038/nm900     Document Type: Article

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