Sense in antisense therapy for spinal muscular atrophy

New England Journal of Medicine

Volumn 366, Issue 8, 2012, Pages 761-763

  MacKenzie, Alex ^a  

^a CHILDREN S HOSPITAL OF EASTERN ONTARIO   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; APOLIPOPROTEIN B; HEMOGLOBIN; MESSENGER RNA; SURVIVAL MOTOR NEURON PROTEIN; SURVIVAL MOTOR NEURON PROTEIN 2; UTROPHIN;

ANTISENSE THERAPY; ARTICLE; CARBOXY TERMINAL SEQUENCE; CLINICAL FEATURE; COPY NUMBER VARIATION; DISEASE SEVERITY; DRUG MEGADOSE; DUCHENNE MUSCULAR DYSTROPHY; GENE CONTROL; GENE DELETION; GENE FUNCTION; GENE PRODUCT; GENETIC DISORDER; HEMOGLOBIN DETERMINATION; HUMAN; IN VITRO STUDY; IN VIVO STUDY; LIFESPAN; MOTONEURON; NEWBORN SCREENING; NONHUMAN; OUTCOME ASSESSMENT; PARESIS; PATHOPHYSIOLOGY; PRIORITY JOURNAL; PROTEIN FUNCTION; PROTEIN INDUCTION; PROTEIN MOTIF; PROTEIN PROCESSING; PROTEIN TARGETING; RNA SPLICING; SMN1 GENE; SPINAL MUSCULAR ATROPHY; THERAPY EFFECT;

EID: 84857396035     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMcibr1114629     Document Type: Article

References (5)

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3. Porensky PN, Mitrpant C, McGovern VL, et al. A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in the mouse. Hum Mol Genet 2011 December 30 (Epub ahead of print).
4. Foust KD, Wang X, McGovern VL, et al. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol 2010;28:271-4.
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