Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice

American Journal of Human Genetics

Volumn 82, Issue 4, 2008, Pages 834-848

  Hua, Yimin ^a   Vickers, Timothy A ^b   Okunola, Hazeem L ^a   Bennett, C Frank ^b   Krainer, Adrian R ^a  

^a Howard Hughes Medical Institute Cold Spring Harbor Laboratory Cold Spring Harbor   (United States)

^b ISIS PHARMACEUTICALS   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CIS ACTING ELEMENT; HETEROGENEOUS NUCLEAR RIBONUCLEOPROTEIN;

AFFINITY CHROMATOGRAPHY; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DISEASE SEVERITY; EXON; FEMALE; GENE; GENE DISRUPTION; HUMAN; HUMAN CELL; INFANT MORTALITY; INTRON; MALE; MOTOR NEURON DISEASE; MOUSE; MUTAGENESIS; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN MOTIF; RNA SPLICING; SILENCER ELEMENT; SMN2 GENE; SPINAL MUSCULAR ATROPHY; TRANSGENIC MOUSE;

ANIMALS; BASE SEQUENCE; CELL LINE; CYCLIC AMP RESPONSE ELEMENT-BINDING PROTEIN; EXONS; GENE THERAPY; HETEROGENEOUS-NUCLEAR RIBONUCLEOPROTEIN GROUP A-B; HUMANS; INTRONS; MICE; MICE, TRANSGENIC; MOLECULAR SEQUENCE DATA; MUSCULAR ATROPHY, SPINAL; NERVE TISSUE PROTEINS; OLIGONUCLEOTIDES, ANTISENSE; RNA SPLICING; RNA-BINDING PROTEINS;

ANIMALIA; MUS MUSCULUS;

EID: 41549168514     PISSN: 00029297     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ajhg.2008.01.014     Document Type: Article

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