Systemic delivery of factor IX messenger RNA for protein replacement therapy

Proceedings of the National Academy of Sciences of the United States of America

Volumn 114, Issue 10, 2017, Pages E1941-E1950

  Ramaswamy, Suvasini ^a   Tonnu, Nina ^a   Tachikawa, Kiyoshi ^b   Limphong, Pattraranee ^b   Vega, Jerel B ^b   Karmali, Priya P ^b   Chivukula, Pad ^b   Verma, Inder M ^a  

^a SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

^b Arcturus   (United States)

Author keywords

Hemophilia B therapy; Hepatic diseases; Lipid nanoparticles; Nonviral mRNA delivery; Systemic delivery

Indexed keywords

BLOOD CLOTTING FACTOR 9; CYTOKINE; LIPID ENABLED AND UNLOCKED NUCLEIC ACID MODIFIED RNA; LIPID NANOPARTICLE; LIVER ENZYME; MESSENGER RNA; NANOPARTICLE; RECOMBINANT BLOOD CLOTTING FACTOR 9; UNCLASSIFIED DRUG; 1,2-DISTEAROYLLECITHIN; CHOLESTEROL; DRUG CARRIER; PHOSPHATIDYLCHOLINE; RECOMBINANT PROTEIN;

ADAPTIVE IMMUNITY; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BLOOD CLOTTING; CONTROLLED STUDY; DRUG DELIVERY SYSTEM; DRUG EFFICACY; FEMALE; HEMOPHILIA B; IMMUNITY; IN VIVO STUDY; INNATE IMMUNITY; LIVER; MALE; MOUSE; NANOENCAPSULATION; NONHUMAN; PRIORITY JOURNAL; PROTEIN REPLACEMENT THERAPY; RECOMMENDED DRUG DOSE; SUBSTITUTION THERAPY; ANIMAL; CHEMISTRY; DISEASE MODEL; DRUG EFFECTS; DRUG FORMULATION; GENE THERAPY; GENETICS; HUMAN; INTRAVENOUS DRUG ADMINISTRATION; KNOCKOUT MOUSE; METABOLISM; PATHOLOGY; PH; PRECLINICAL STUDY; PROCEDURES; SECRETION (PROCESS);

ANIMALS; CHOLESTEROL; CYTOKINES; DISEASE MODELS, ANIMAL; DRUG CARRIERS; DRUG COMPOUNDING; DRUG EVALUATION, PRECLINICAL; FACTOR IX; FEMALE; GENETIC THERAPY; HEMOPHILIA B; HUMANS; HYDROGEN-ION CONCENTRATION; INJECTIONS, INTRAVENOUS; LIVER; MALE; MICE; MICE, KNOCKOUT; NANOPARTICLES; PHOSPHATIDYLCHOLINES; RECOMBINANT PROTEINS; RNA, MESSENGER;

EID: 85014634181     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1619653114     Document Type: Article

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