A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver

Molecular Therapy Nucleic Acids

Volumn 3, Issue , 2014, Pages e193-

  Gallego Villar, Lorena ^a   Viecelli, Hiu Man ^b   Pérez, Belén ^a   Harding, Cary O ^c,d   Ugarte, Magdalena ^a   Thöny, Beat ^b   Desviat, Lourdes R ^a  

^a UNIVERSIDAD AUTÓNOMA DE MADRID   (Spain)

^b UNIVERSITY OF ZURICH   (Switzerland)

^c OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^d OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

Author keywords

Animal models; Antisense oligonucleotides; Exon skipping; Hyperphenylalaninemia; Metabolic diseases; Splicing suppression; Vivo morpholino

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; LOCKED NUCLEIC ACID; MESSENGER RNA PRECURSOR; MORPHOLINO OLIGONUCLEOTIDE; PHENYLALANINE; PHENYLALANINE 4 MONOOXYGENASE; UNCLASSIFIED DRUG; VIVO MORPHOLINO;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANTISENSE THERAPY; ARTICLE; CONTROLLED STUDY; ENZYME ACTIVITY; EXON SKIPPING; GENE EXPRESSION; GENE MUTATION; GENETIC DISORDER; HEPATOMA CELL; HETEROZYGOTE; HUMAN; HUMAN CELL; HYPERPHENYLALANINEMIA; IN VITRO STUDY; IN VIVO STUDY; KIDNEY; LIVER; LIVER DISEASE; LIVER TOXICITY; MOUSE; NONHUMAN; PRIORITY JOURNAL; QUANTITATIVE ANALYSIS; TANDEM MASS SPECTROMETRY; WILD TYPE;

EID: 84925235142     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2014.44     Document Type: Article

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