RNA-targeted splice-correction therapy for neuromuscular disease

Brain

Volumn 133, Issue 4, 2010, Pages 957-972

  Wood, Matthew J A ^a   Gait, Michael J ^b   Yin, Haifang ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b MRC LABORATORY OF MOLECULAR BIOLOGY   (United Kingdom)

Author keywords

Antisense oligonucleotide; Dystrophin; Exon skipping; RNA splicing

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ATALUREN; ATAXIN 1; ATM PROTEIN; BETA ADRENERGIC RECEPTOR BLOCKING AGENT; BETA GLOBIN; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; DYSTROPHIN; FRAGILE X MENTAL RETARDATION PROTEIN; GLUCOCORTICOID; MESSENGER RNA; MESSENGER RNA PRECURSOR; MORPHOLINO PHOSPHORODIAMIDATE; NEUROFIBROMIN; SURVIVAL MOTOR NEURON PROTEIN; UNCLASSIFIED DRUG; UTROPHIN;

ADENOVIRUS; AMYOTROPHIC LATERAL SCLEROSIS; ATAXIA TELANGIECTASIA; BETA THALASSEMIA; CARDIOMYOPATHY; CAUSE OF DEATH; CHROMOSOME 17; CORTICOSTEROID THERAPY; DUCHENNE MUSCULAR DYSTROPHY; ELECTROPHYSIOLOGY; EXON; FRAGILE X SYNDROME; FRONTOTEMPORAL DEMENTIA; GENE CONTROL; GENE DELETION; GENE MUTATION; GENE REPLACEMENT THERAPY; GENE TARGETING; GENE THERAPY; GENETIC HETEROGENEITY; GENETIC TRANSCRIPTION; GOLD STANDARD; HEARING LOSS; HEART FAILURE; HIGH THROUGHPUT SCREENING; LIFE EXPECTANCY; LOSS OF FUNCTION MUTATION; MOLECULAR THERAPY; NEUROMUSCULAR DISEASE; NEWBORN; NONHUMAN; OPEN READING FRAME; PARKINSONISM; POINT MUTATION; PRADER WILLI SYNDROME; PRIORITY JOURNAL; PROGRESSIVE MUSCULAR DYSTROPHY; PROTEIN EXPRESSION; RESPIRATORY FAILURE; REVIEW; RNA SPLICING; RNA TRANSLATION; SPINAL MUSCULAR ATROPHY; SYSTEMIC THERAPY; UPREGULATION; VIRAL GENE DELIVERY SYSTEM; VIRUS GENOME;

EID: 77950793623     PISSN: 00068950     EISSN: 14602156     Source Type: Journal    
DOI: 10.1093/brain/awq002     Document Type: Review

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