Gene therapy strategies for X-linked myotubular myopathy
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DOI: 10.1080/21678707.2018.1443807
Abstract: ABSTRACT Introduction: X-linked myotubular myopathy (XLMTM) is a severe, frequently fatal, type of congenital myopathy for which only supportive care is currently available. XLMTM is due to MTM1 mutations that lead to a deficiency in… read more here.
Keywords: gene therapy; linked myotubular; gene; xlmtm ... See more keywords