A gene therapeutic approach to correct splice defects with modified U1 and U6 snRNPs

Human Gene Therapy

Volumn 24, Issue 1, 2013, Pages 97-104

  Schmid, Fabian ^a   Hiller, Thomas ^a   Korner, Germaine ^a   Glaus, Esther ^a   Berger, Wolfgang ^a,b   Neidhardt, John ^a,c  

^a UNIVERSITY OF ZURICH   (Switzerland)

^b Neuroscience Center Zurich and Division of Neurosurgery   (Switzerland)

^c Life Science Zurich   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

RNA; SMALL NUCLEAR RIBONUCLEOPROTEIN;

ADAPTATION; ARTICLE; CONTROLLED STUDY; EXON; GENE MUTATION; GENE THERAPY; GENETIC TRANSCRIPTION; HUMAN; HUMAN CELL; INTRON; RNA SPLICING; SKIN FIBROBLAST;

ALTERNATIVE SPLICING; ANIMALS; CERCOPITHECUS AETHIOPS; COS CELLS; GENETIC THERAPY; HUMANS; MICROTUBULE-ASSOCIATED PROTEINS; MUTAGENESIS, SITE-DIRECTED; MUTATION; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RIBONUCLEOPROTEIN, U1 SMALL NUCLEAR; RIBONUCLEOPROTEIN, U4-U6 SMALL NUCLEAR; RNA SPLICE SITES; TREATMENT OUTCOME;

EID: 84872794005     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2012.110     Document Type: Article

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