Stimulating Full-Length SMN2 Expression by Delivering Bifunctional RNAs via a Viral Vector

Molecular Therapy

Volumn 14, Issue 1, 2006, Pages 54-62

  Baughan, Travis ^a   Shababi, Monir ^b   Coady, Tristan H ^b   Dickson, Alexa M ^a   Tullis, Gregory E ^a   Lorson, Christian L ^a,b  

^a University of Missouri   (United States)

^b UNIVERSITY OF MISSOURI   (United States)

Author keywords

alternative splicing; gene therapy; SMN1 gene product; SMN2 gene product; spinal muscular atrophy

Indexed keywords

COMPLEMENTARY RNA; ISOPROTEIN; PARVOVIRUS VECTOR; SURVIVAL MOTOR NEURON PROTEIN; VIRUS VECTOR;

ARTICLE; AUTOSOMAL RECESSIVE DISORDER; CONTROLLED STUDY; DRUG TARGETING; EXON; FIBROBLAST; GENE LOSS; HUMAN; HUMAN CELL; IN VIVO STUDY; INFANT MORTALITY; PLASMID; PROTEIN BINDING; PROTEIN DOMAIN; PROTEIN EXPRESSION; PROTEIN FUNCTION; PROTEIN PROCESSING; RNA SEQUENCE; SINGLE NUCLEOTIDE POLYMORPHISM; SPINAL MUSCULAR ATROPHY; VIRAL GENE DELIVERY SYSTEM;

BLOTTING, WESTERN; CELLS, CULTURED; CYCLIC AMP RESPONSE ELEMENT-BINDING PROTEIN; DEPENDOVIRUS; EXONS; FIBROBLASTS; GENETIC VECTORS; HELA CELLS; HUMANS; LUCIFERASES; MICROSCOPY, FLUORESCENCE; MODELS, GENETIC; MUSCULAR ATROPHY, SPINAL; NERVE TISSUE PROTEINS; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA; RNA PRECURSORS; RNA SPLICING; RNA-BINDING PROTEINS; TRANSFECTION;

ADENO-ASSOCIATED VIRUS;

EID: 33745144314     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2006.01.012     Document Type: Article

References (39)

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