• Publications
  • Influence
SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
Drugs that provide the splice of life Motor neurons relay signals from the nervous system to muscle fibers. In patients with spinal muscular atrophy, a protein required for the survival of theseExpand
  • 248
  • 13
The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential
Nonsense mutations, resulting in a premature stop codon in the open reading frame of mRNAs are responsible for thousands of inherited diseases. Readthrough of premature stop codons by small moleculeExpand
  • 10
  • 1
Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy.
Spinal muscular atrophy (SMA) is the leading genetic cause of infant and toddler mortality, and there is currently no approved therapy available. SMA is caused by mutation or deletion of the survivalExpand
  • 35
Naryshkin mice with spinal muscular atrophy splicing modifiers improve motor function and longevity in SMN 2
clicking here. colleagues, clients, or customers by , you can order high-quality copies for your If you wish to distribute this article to others here. following the guidelines can be obtained byExpand