Restoration of SMN function: Delivery of a trans-splicing RNA Re-directs SMN2 pre-mRNA splicing

Molecular Therapy

Volumn 15, Issue 8, 2007, Pages 1471-1478

  Coady, Tristan H ^a   Shababi, Monir ^a   Tullis, Gregory E ^b   Lorson, Christian L ^a  

^a UNIVERSITY OF MISSOURI   (United States)

^b BOSTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

MESSENGER RNA; MESSENGER RNA PRECURSOR; PARVOVIRUS VECTOR; RNA; SMALL NUCLEAR RIBONUCLEOPROTEIN; SURVIVAL MOTOR NEURON PROTEIN; SURVIVAL MOTOR NEURON PROTEIN 2; UNCLASSIFIED DRUG;

ALTERNATIVE RNA SPLICING; ARTICLE; CELL STRAIN HEK293; CONTROLLED STUDY; DISEASE SEVERITY; EXON; FIBROBLAST; GENE LOSS; GENE TARGETING; HELA CELL; HUMAN; HUMAN CELL; PATHOGENESIS; PROTEIN EXPRESSION; PROTEIN FUNCTION; RNA SEQUENCE; RNA SPLICING; SPINAL MUSCULAR ATROPHY; TRANS SPLICING; VIRAL GENE DELIVERY SYSTEM; VIRUS RECOMBINANT;

ALTERNATIVE SPLICING; BASE SEQUENCE; CELL LINE; CELL SURVIVAL; CYCLIC AMP RESPONSE ELEMENT-BINDING PROTEIN; FIBROBLASTS; GENE TRANSFER TECHNIQUES; HUMANS; MOLECULAR SEQUENCE DATA; MUSCULAR ATROPHY, SPINAL; NERVE TISSUE PROTEINS; RNA, MESSENGER; RNA-BINDING PROTEINS; TRANS-SPLICING; TRANSCRIPTION, GENETIC;

EID: 34547117851     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/sj.mt.6300222     Document Type: Article

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