Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides

Nature Biotechnology

Volumn 35, Issue 9, 2017, Pages 845-851

  Iwamoto, Naoki ^a,d   Butler, David C D ^a,d   Svrzikapa, Nenad ^a,d   Mohapatra, Susovan ^a   Zlatev, Ivan ^c   Sah, DInah W Y ^a,c   Meena, ^a   Standley, Stephany M ^a   Lu, Genliang ^a   Apponi, Luciano H ^a   Frank Kamenetsky, Maria ^a   Zhang, Jason Jingxin ^a   Vargeese, Chandra ^a   Verdine, Gregory L ^a,b  

^a currently at Wave Life Sciences   (United States)

^b Harvard University   (United States)

^c ALNYLAM PHARMACEUTICALS   (United States)

^d VOYAGER THERAPEUTICS   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ISOMERS; OLIGONUCLEOTIDES; STEREOSELECTIVITY; SYNTHESIS (CHEMICAL);

ANTISENSE OLIGONUCLEOTIDES; CHEMICAL PURITY; IN-VITRO; PHOSPHOROTHIOATES; RNA CLEAVAGE; SMALL MOLECULES; STEREOCHEMICAL PURITY; SYNTHETIC PROCESS;

STEREOCHEMISTRY;

DNA; MESSENGER RNA; MIPOMERSEN; PHOSPHOROTHIOIC ACID; ANTISENSE OLIGONUCLEOTIDE; OLIGONUCLEOTIDE; OLIGONUCLEOTIDE PHOSPHOROTHIOATE; RIBONUCLEASE H; RIBONUCLEASE HI;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; CRYSTAL STRUCTURE; DRUG DEGRADATION; DRUG DESIGN; DRUG DOSE TITRATION; DRUG EFFICACY; DRUG HALF LIFE; DRUG INACTIVATION; DRUG PURITY; DRUG STABILITY; DRUG SYNTHESIS; HUMAN; LIPOPHILICITY; LIVER HOMOGENATE; MALE; MELTING TEMPERATURE; NONHUMAN; NUCLEOPHILICITY; PLASMA PROTEIN BINDING; PRIORITY JOURNAL; RAT; RETENTION TIME; REVERSED PHASE HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; RNA CLEAVAGE; STEREOCHEMISTRY; STEREOISOMERISM; THERMOSTABILITY; ANIMAL; C57BL MOUSE; CHEMICAL PHENOMENA; CHEMISTRY; FEMALE; GENE THERAPY; METABOLISM; MOUSE; PROCEDURES; SPRAGUE DAWLEY RAT; TRANSGENIC MOUSE;

ANIMALS; DRUG STABILITY; FEMALE; GENETIC THERAPY; HUMANS; HYDROPHOBIC AND HYDROPHILIC INTERACTIONS; MALE; MICE; MICE, INBRED C57BL; MICE, TRANSGENIC; OLIGONUCLEOTIDES; OLIGONUCLEOTIDES, ANTISENSE; PHOSPHOROTHIOATE OLIGONUCLEOTIDES; RATS; RATS, SPRAGUE-DAWLEY; RIBONUCLEASE H; STEREOISOMERISM;

EID: 85029279170     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt.3948     Document Type: Article

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