Development of 2′-O-Methyl and LNA Antisense Oligonucleotides for <i>SMN2</i> Splicing Correction in SMA Cells

Eitemau Tebyg

• Assessment of Nuclear Gem Quantity for Evaluating the Efficacy of Antisense Oligonucleotides in Spinal Muscular Atrophy Cells
  gan: Haya Al-Hilal, et al.
  Cyhoeddwyd: (2024-01-01)
• Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy
  gan: Tejal Aslesh, et al.
  Cyhoeddwyd: (2022-01-01)
• The Relevance of Spinal Muscular Atrophy Biomarkers in the Treatment Era
  gan: Marianna Maretina, et al.
  Cyhoeddwyd: (2024-10-01)
• Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
  gan: Audrey M. Winkelsas, et al.
  Cyhoeddwyd: (2021-03-01)
• Combinatorial ASO-mediated therapy with low dose SMN and the protective modifier Chp1 is not sufficient to ameliorate SMA pathology hallmarks
  gan: A. Muinos-Bühl, et al.
  Cyhoeddwyd: (2022-09-01)