Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases

Trends in Molecular Medicine

Volumn 18, Issue 11, 2012, Pages 634-643

  Southwell, Amber L ^a   Skotte, Niels H ^a   Bennett, C Frank ^b   Hayden, Michael R ^a  

^a UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^b ISIS PHARMACEUTICALS   (United States)

Author keywords

Alzheimer disease; Amyotrophic lateral sclerosis; Gene silencing; Gene therapy; Huntington disease; Spinal muscular atrophy; Splice modification

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; HUNTINGTIN; MESSENGER RNA; MORPHOLINO OLIGONUCLEOTIDE; SMALL INTERFERING RNA;

ALTERNATIVE RNA SPLICING; ALZHEIMER DISEASE; AMYOTROPHIC LATERAL SCLEROSIS; ANTISENSE THERAPY; CENTRAL NERVOUS SYSTEM; CHILL; CHROMOSOME 17; DISEASE COURSE; DNA MODIFICATION; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG SAFETY; FEVER; FRONTOTEMPORAL DEMENTIA; GENE FUNCTION; GENE INACTIVATION; GENE MUTATION; GENE PRODUCT; GENE SILENCING; GENE TARGETING; HUMAN; HUNTINGTON CHOREA; INHERITANCE; NAUSEA; NONHUMAN; PARKINSONISM; PHASE 1 CLINICAL TRIAL (TOPIC); REVIEW; RNA INTERFERENCE; RNA METABOLISM; SPINAL MUSCULAR ATROPHY;

ANIMALS; CLINICAL TRIALS AS TOPIC; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; HEREDODEGENERATIVE DISORDERS, NERVOUS SYSTEM; HUMANS; OLIGONUCLEOTIDES, ANTISENSE; RNA INTERFERENCE;

EID: 84868208876     PISSN: 14714914     EISSN: 1471499X     Source Type: Journal    
DOI: 10.1016/j.molmed.2012.09.001     Document Type: Review

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