Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system

Gene Therapy

Volumn 23, Issue 10, 2016, Pages 699-707

  DeRosa, F ^a   Guild, B ^a   Karve, S ^a   Smith, L ^a   Love, K ^b   Dorkin, J R ^b   Kauffman, K J ^b,c   Zhang, J ^a   Yahalom, B ^d   Anderson, D G ^b,c,e   Heartlein, M W ^a  

^a Shire Pharmaceuticals   (United States)

^b MASSACHUSETTS INSTITUTE OF TECHNOLOGY   (United States)

^c Massachusetts Institute of Technology   (United States)

^d Biomedical Research Models Inc   (United States)

^e HARVARD MIT DIVISION OF HEALTH SCIENCES AND TECHNOLOGY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ERYTHROPOIETIN; MESSENGER RNA; RECOMBINANT BLOOD CLOTTING FACTOR 9; RECOMBINANT ERYTHROPOIETIN; BLOOD CLOTTING FACTOR 9; NANOPARTICLE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG FORMULATION; GENETIC TRANSCRIPTION; HEMATOCRIT; HEMOPHILIA B; IN VIVO STUDY; MACACA FASCICULARIS; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; SINGLE DRUG DOSE; SUSTAINED DRUG RELEASE; ADVERSE EFFECTS; ANIMAL; BLOOD; C57BL MOUSE; CHEMISTRY; GENE THERAPY; GENETICS; LIVER; METABOLISM; PROCEDURES;

ANIMALS; ERYTHROPOIETIN; FACTOR IX; GENETIC THERAPY; HEMATOCRIT; HEMOPHILIA B; LIVER; MALE; MICE; MICE, INBRED C57BL; NANOPARTICLES; RNA, MESSENGER;

EID: 84976580479     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2016.46     Document Type: Article

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