Spinal Muscular Atrophy: From Gene to Therapy

Seminars in Pediatric Neurology

Volumn 13, Issue 2, 2006, Pages 121-131

  Wirth, Brunhilde ^a   Brichta, Lars ^a   Hahnen, Eric ^a  

^a UNIVERSITY OF COLOGNE   (Germany)

Author keywords

neuromuscular disorder; SMA therapy; spinal muscular atrophy; survival motor neuron; valproic acid

Indexed keywords

4 PHENYLBUTYRIC ACID; 5 AZA 2' DEOXYCYTIDINE; ACLARUBICIN; AMINOGLYCOSIDE DERIVATIVE; BUTYRIC ACID; CARDIOTROPHIN 1; COMPLEMENTARY RNA; HISTONE DEACETYLASE; HISTONE DEACETYLASE INHIBITOR; HYDROXYUREA; INDOPROFEN; INTERFERON; M 344; NEUROPROTECTIVE AGENT; NEUROTROPHIC FACTOR; PHOSPHATASE; PHOSPHOTRANSFERASE; RILUZOLE; SURVIVAL MOTOR NEURON PROTEIN; UNCLASSIFIED DRUG; VALPROIC ACID; VANADATE SODIUM; VORINOSTAT;

CLINICAL TRIAL; DISEASE CARRIER; DISEASE SEVERITY; DNA METHYLATION; DRUG EFFECT; DRUG MECHANISM; EXERCISE; EXON; GENE; GENE ACTIVATION; GENE CONVERSION; GENE DELETION; GENE MUTATION; GENE THERAPY; GENETIC CODE; GENETIC TRANSCRIPTION; HETEROZYGOSITY; HUMAN; IN VIVO STUDY; MODULATION; MOLECULAR GENETICS; MOTOR PERFORMANCE; NEWBORN SCREENING; NONHUMAN; PRENATAL DIAGNOSIS; PROMOTER REGION; PROTEIN FUNCTION; PROTEIN STABILITY; REVIEW; RNA SPLICING; SILENT GENE; SPINAL MUSCULAR ATROPHY; STEM CELL TRANSPLANTATION; TRANSCRIPTION INITIATION;

ANIMALS; CYCLIC AMP RESPONSE ELEMENT-BINDING PROTEIN; HUMANS; MODELS, MOLECULAR; MUSCULAR ATROPHY, SPINAL; MUTATION; NERVE TISSUE PROTEINS; RNA-BINDING PROTEINS; SMN COMPLEX PROTEINS; SURVIVAL OF MOTOR NEURON 1 PROTEIN; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

EID: 33749264614     PISSN: 10719091     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.spen.2006.06.008     Document Type: Review

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