Biblio
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial. Lancet Neurol. 2023;22(12):1125-1139.
Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs. Ann Transl Med. 2015;3(17):257.
. Gene therapy in monogenic congenital myopathies. Methods. 2015.
. Muscle pathology, limb strength, walking gait, respiratory function and neurological impairment establish disease progression in the p.N155K canine model of X-linked myotubular myopathy. Ann Transl Med. 2015;3(18):262.
. Use of Adeno-Associated Virus to Enrich Cardiomyocytes Derived from Human Stem Cells. Hum Gene Ther Clin Dev. 2015.
. Disease-in-a-Dish: The Contribution of Patient-Specific Induced Pluripotent Stem Cell Technology to Regenerative Rehabilitation. Am J Phys Med Rehabil. 2014.
. Gait characteristics in a canine model of X-linked myotubular myopathy. J Neurol Sci. 2014.
. Gene Therapy for Inherited Muscle Diseases: Where Genetics Meets Rehabilitation Medicine. Am J Phys Med Rehabil. 2014;93(11 Suppl 3):S97-S107.
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