Antisense oligonucleotide-based therapies for diseases caused by pre-mRNA processing defects

Advances in Experimental Medicine and Biology

Volumn 825, Issue , 2014, Pages 303-352

  Rigo, Frank ^a   Seth, Punit P ^a   Bennett, C Frank ^a  

^a ISIS PHARMACEUTICALS   (United States)

Author keywords

Antisense oligonucleotide; Fukuyama congenital muscular dystrophy; Hutchinson Gilford progeria syndrome; Myotonic dystrophy; Pre mRNA processing; RNA binding protein; RNase H; siRNA; Spinal muscular atrophy; Splicing; Usher syndrome

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ANTISENSE OLIGONUCLEOTIDE 10 27; DEOXYRIBOZYME; DOUBLE STRANDED RNA; ETEPLIRSEN; MESSENGER RNA; MICRORNA; MORPHOLINO OLIGONUCLEOTIDE; POLYCOMB REPRESSIVE COMPLEX 2; RIBONUCLEASE H; RIBOZYME; RNA BINDING PROTEIN; SINGLE STRANDED RNA; SMALL INTERFERING RNA; UNCLASSIFIED DRUG; RNA PRECURSOR;

ARTICLE; BINDING AFFINITY; DRUG DESIGN; DRUG DISTRIBUTION; DRUG MECHANISM; DRUG RESPONSE; DRUG TRANSPORT; FUKUYAMA CONGENITAL MUSCULAR DYSTROPHY; GENE EXPRESSION; GENE LOCATION; HUMAN; HYDROGEN BOND; MOLECULAR MIMICRY; MOLECULARLY TARGETED THERAPY; MYOTONIC DYSTROPHY; NONHUMAN; POLYADENYLATION; PRIORITY JOURNAL; PROGERIA; PROTEIN SECONDARY STRUCTURE; RNA BINDING; RNA CLEAVAGE; RNA DEGRADATION; RNA EDITING; RNA PROCESSING; RNA RECOMBINATION; RNA SPLICING; SPINAL MUSCULAR ATROPHY; TOXICITY TESTING; UPREGULATION; USHER SYNDROME; ANIMAL; DISEASE MODEL; GENETIC DISEASES, INBORN; GENETICS; METABOLISM;

ANIMALIA;

ANIMALS; DISEASE MODELS, ANIMAL; GENETIC DISEASES, INBORN; HUMANS; OLIGONUCLEOTIDES, ANTISENSE; RNA PRECURSORS; RNA PROCESSING, POST-TRANSCRIPTIONAL;

EID: 84921996531     PISSN: 00652598     EISSN: 22148019     Source Type: Book Series    
DOI: 10.1007/978-1-4939-1221-6_9     Document Type: Article

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