RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice

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

Volumn 111, Issue 21, 2014, Pages 7777-7782

  Yasuda, Makiko ^a   Gan, Lin ^a   Chen, Brenden ^a   Kadirvel, Senkottuvelan ^a   Yu, Chunli ^a   Phillips, John D ^b   New, Maria I ^a   Liebow, Abigail ^c   Fitzgerald, Kevin ^c   Querbes, William ^c   Desnick, Robert J ^a  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^c ALNYLAM PHARMACEUTICALS   (United States)

Author keywords

Heme biosynthetic disorders; Liver targeted siRNA; RNAi therapeutics

Indexed keywords

5 AMINOLEVULINATE SYNTHASE; 5 AMINOLEVULINATE SYNTHASE 1; ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; CYTOCHROME P450 2E1; HEMIN; MESSENGER RNA; PHENOBARBITAL; PORPHOBILINOGEN; SMALL INTERFERING RNA; SOLID LIPID NANOPARTICLE; UNCLASSIFIED DRUG;

ACUTE INTERMITTENT PORPHYRIA; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; CONTROLLED STUDY; DOSE RESPONSE; DRUG TOLERABILITY; ENZYME ACTIVITY; FEMALE; GENE EXPRESSION; GENE SILENCING; GENE TARGETING; HEPATIC PORPHYRIA; LIVER CELL; MALE; MOTONEURON; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; RNA INTERFERENCE; SINGLE DRUG DOSE; TRYPTOPHAN METABOLISM; UPREGULATION;

HEME BIOSYNTHETIC DISORDERS; LIVER-TARGETED SIRNA; RNAI THERAPEUTICS;

5-AMINOLEVULINATE SYNTHETASE; ANALYSIS OF VARIANCE; ANIMALS; BLOTTING, WESTERN; DRUG EVALUATION, PRECLINICAL; ELECTROPHORESIS, POLYACRYLAMIDE GEL; FEMALE; LIVER; MICE; MICE, INBRED C57BL; PARTICLE SIZE; PORPHYRIA, ACUTE INTERMITTENT; REAL-TIME POLYMERASE CHAIN REACTION; RNA INTERFERENCE; RNA, SMALL INTERFERING;

EID: 84901660512     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1406228111     Document Type: Article

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