Genetic therapies for RNA mis-splicing diseases

Trends in Genetics

Volumn 27, Issue 5, 2011, Pages 196-205

  Hammond, Suzan M ^a   Wood, Matthew J A ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; NUCLEAR RNA; RNA;

ATAXIA TELANGIECTASIA; ATHEROSCLEROSIS; BETA THALASSEMIA; CONGENITAL DISORDER OF GLYCOSYLATION; DUCHENNE MUSCULAR DYSTROPHY; DYSFERLINOPATHY; EXON SKIPPING; GENETIC DISORDER; HUMAN; METHYLMALONIC ACIDURIA; MUTATION; NEUROFIBROMATOSIS; NIEMANN PICK DISEASE; NONHUMAN; OCULAR ALBINISM; OLIGONUCLEOTIDE THERAPY; PARKINSONISM; PRIORITY JOURNAL; PROGERIA; PROPIONIC ACIDEMIA; PROTEIN MODIFICATION; REVIEW; RNA MIS SPLICING DISEASE; RNA SPLICING; SPINAL MUSCULAR ATROPHY; TRANS SPLICING;

ANIMALS; DNA, ANTISENSE; EXONS; GENE THERAPY; HUMANS; MUTATION; OLIGONUCLEOTIDES; RNA SPLICING;

EID: 79955093434     PISSN: 01689525     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.tig.2011.02.004     Document Type: Review

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