Antisense oligonucleotide mediated therapy of spinal muscular atrophy

Translational Neuroscience

Volumn 4, Issue 1, 2013, Pages 1-7

  Sivanesan, Senthilkumar ^a   Howell, Matthew D ^a   Didonato, Christine J ^b,c   Singh, Ravindra N ^a  

^a IOWA STATE UNIVERSITY   (United States)

^b NORTHWESTERN UNIVERSITY   (United States)

^c CHILDREN'S MEMORIAL HOSPITAL   (United States)

Author keywords

Antisense oligonucleotide; ASO; ISS N1; MOE; PMO; SMA; SMN; Splicing

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; MORPHOLINO OLIGONUCLEOTIDE; SURVIVAL MOTOR NEURON PROTEIN 2;

ANTISENSE THERAPY; DOSE RESPONSE; DRUG EFFICACY; DRUG MEGADOSE; DRUG TARGETING; DRUG TOLERABILITY; EXON; INTRONIC SPLICING SILENCER N1; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; REVIEW; RNA SPLICING; SILENCER ELEMENT; SINGLE DRUG DOSE; SPINAL MUSCULAR ATROPHY; SURVIVAL MOTOR NEURON PROTEIN 2 GENE;

EID: 84874915156     PISSN: 20813856     EISSN: 20816936     Source Type: Journal    
DOI: 10.2478/s13380-013-0109-2     Document Type: Review

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