Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in vivo

Biomaterials

Volumn 109, Issue , 2016, Pages 78-87

  Kauffman, Kevin J ^a,b   Mir, Faryal F ^b   Jhunjhunwala, Siddharth ^b   Kaczmarek, James C ^a,b   Hurtado, Juan E ^a,b   Yang, Jung H ^b   Webber, Matthew J ^b   Kowalski, Piotr S ^b   Heartlein, Michael W ^c   DeRosa, Frank ^c   Anderson, Daniel G ^a,b  

^a USA   (United States)

^b MASSACHUSETTS INSTITUTE OF TECHNOLOGY   (United States)

^c Shire Pharmaceuticals   (United States)

Author keywords

Base modification; Immmune response; In vivo; Lipid nanoparticle; mRNA; Pseudouridine

Indexed keywords

ENZYME ACTIVITY; IMMUNE SYSTEM; RNA;

BASE MODIFICATION; IMMMUNE RESPONSE; LIPID NANOPARTICLES; MRNA; PSEUDOURIDINE;

NANOPARTICLES;

LIPID NANOPARTICLE; MESSENGER RNA; NANOPARTICLE; PSEUDOURIDINE; UNCLASSIFIED DRUG; CYTOKINE; LIPID;

ANIMAL EXPERIMENT; ARTICLE; BONE MARROW CELL; CELL ACTIVATION; CONTROLLED STUDY; CYTOKINE RELEASE; DRUG DELIVERY SYSTEM; DRUG EFFICACY; FEMALE; IMMUNE RESPONSE; IMMUNOGENICITY; IN VITRO GENE TRANSFER; IN VIVO GENE TRANSFER; IN VIVO STUDY; INNATE IMMUNITY; MOUSE; NEUTROPHILIA; NONHUMAN; PHYSICAL CHEMISTRY; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; PROTEIN EXPRESSION; UPREGULATION; ANIMAL; BIOSYNTHESIS; C57BL MOUSE; CHEMISTRY; GENE EXPRESSION; GENE TRANSFER; GENETIC TRANSFECTION; HELA CELL LINE; HUMAN; IMMUNOLOGY; LIVER; METABOLISM; PARTICLE SIZE; SURFACE PROPERTY;

ANIMALS; CYTOKINES; FEMALE; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; HELA CELLS; HUMANS; IMMUNITY, INNATE; LIPIDS; LIVER; MICE, INBRED C57BL; MYELOID CELLS; NANOPARTICLES; PARTICLE SIZE; PSEUDOURIDINE; RNA, MESSENGER; SURFACE PROPERTIES; TRANSFECTION;

EID: 84988663801     PISSN: 01429612     EISSN: 18785905     Source Type: Journal    
DOI: 10.1016/j.biomaterials.2016.09.006     Document Type: Article

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