Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs

Eitemau Tebyg

• Enzyme replacement therapy rescues weakness and improves muscle pathology in mice with X-linked myotubular myopathy
  gan: Lawlor, Michael W., et al.
  Cyhoeddwyd: (2013)
• Phosphatidylinositol 3-kinase inhibition restores Ca(2+) release defects and prolongs survival in myotubularin-deficient mice
  gan: Kutchukian, Candice, et al.
  Cyhoeddwyd: (2016)
• Inhibition of Activin Receptor Type IIB Increases Strength and Lifespan in Myotubularin-Deficient Mice
  gan: Lawlor, Michael W., et al.
  Cyhoeddwyd: (2011)
• Myotubularin-Deficient Myoblasts Display Increased Apoptosis, Delayed Proliferation, and Poor Cell Engraftment
  gan: Lawlor, Michael W., et al.
  Cyhoeddwyd: (2012)
• T-tubule disorganization and defective excitation-contraction coupling in muscle fibers lacking myotubularin lipid phosphatase
  gan: Al-Qusairi, Lama, et al.
  Cyhoeddwyd: (2009)