Spinal Muscular Atrophy Therapeutics: Where do we Stand?

This publication has 109 references indexed in Scilit:

• Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy
  PLOS ONE, 2013
• Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of Spinal Muscular Atrophy
  Biochemical and Biophysical Research Communications, 2012
• Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders
  Molecular Therapy, 2011
• Muscles in a mouse model of spinal muscular atrophy show profound defects in neuromuscular development even in the absence of failure in neuromuscular transmission or loss of motor neurons
  Developmental Biology, 2011
• Early Functional Impairment of Sensory-Motor Connectivity in a Mouse Model of Spinal Muscular Atrophy
  Neuron, 2011
• RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
  Nature Biotechnology, 2010
• DcpS as a Therapeutic Target for Spinal Muscular Atrophy
  ACS Chemical Biology, 2008
• SMN Deficiency Causes Tissue-Specific Perturbations in the Repertoire of snRNAs and Widespread Defects in Splicing
  Cell, 2008
• Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice
  The American Journal of Human Genetics, 2008
• The language of covalent histone modifications
  Nature, 2000