Hyperactive PiggyBac transposons for sustained and robust liver-targeted gene therapy

Molecular Therapy

Volumn 22, Issue 9, 2014, Pages 1614-1624

  Di Matteo, Mario ^a,b   Samara Kuko, Emira ^a   Ward, Natalie J ^c   Waddingon, Simon N ^d,e   Mcvey, John H ^f   Kl Chuah, Marinee ^a,b   VandenDriessche, Thierry ^a,b  

^a FREE UNIVERSITY OF BRUSSELS   (Belgium)

^b UNIVERSITY OF LEUVEN   (Belgium)

^c UNIVERSITY COLLEGE LONDON   (United Kingdom)

^d UNIVERSITY COLLEGE LONDON   (United Kingdom)

^e UNIVERSITY OF THE WITWATERSRAND   (South Africa)

^f UNIVERSITY OF SURREY   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; MESSENGER RNA; TRANSPOSASE; TRANSPOSON;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CANCER RISK; CARCINOGENESIS; CODON USAGE; CONTROLLED STUDY; DNA MODIFICATION; DRUG POTENCY; DRUG SAFETY; GENE DELIVERY SYSTEM; GENE DOSAGE; GENE EXPRESSION; GENE STRUCTURE; GENE THERAPY; GENE TRANSFER; GENETIC TRANSFECTION; HEMOPHILIA B; IMMUNOLOGICAL TOLERANCE; INSERTIONAL ONCOGENESIS; LIVER CELL; MOUSE; MUTATIONAL ANALYSIS; NONHUMAN; PHENOTYPE; PIGGYBAC TRANSPOSON; PROCESS OPTIMIZATION; PROMOTER REGION; PROTEIN EXPRESSION; PROTEIN SECRETION; PROTEIN STABILITY; RISK ASSESSMENT; ROBUST LIVER TARGETED GENE THERAPY; SURVIVAL RATE; SURVIVAL TIME; SUSTAINED LIVER TARGETED GENE THERAPY; TRANSPOSON; TRANSPOSON TERMINAL REPEAT; VALIDATION PROCESS; ADMINISTRATION AND DOSAGE; ANIMAL; ANTIBODY SPECIFICITY; C57BL MOUSE; DISEASE MODEL; GENE VECTOR; GENETICS; HUMAN; IMMUNOLOGY; METABOLISM; PATHOLOGY; PROCEDURES; THERAPEUTIC USE;

ANIMALS; DISEASE MODELS, ANIMAL; DNA TRANSPOSABLE ELEMENTS; FACTOR IX; GENETIC THERAPY; GENETIC VECTORS; HEMOPHILIA B; HEPATOCYTES; HUMANS; MICE; MICE, INBRED C57BL; ORGAN SPECIFICITY; TRANSPOSASES;

EID: 84988349784     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2014.131     Document Type: Article

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