Gene therapy in monogenic congenital myopathies.
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ABSTRACT:
SUBMITTER: Guan X
PROVIDER: S-EPMC4826856 | biostudies-literature | 2016 Apr
REPOSITORIES: biostudies-literature
Publications
Gene therapy in monogenic congenital myopathies.
Guan Xuan X Goddard Melissa A MA Mack David L DL Childers Martin K MK
Methods (San Diego, Calif.) 20151014
Current treatment options for patients with monogenetic congenital myopathies (MCM) ameliorate the symptoms of the disorder without resolving the underlying cause. However, gene therapies are being developed where the mutated or deficient gene target is replaced. Preclinical findings in animal models appear promising, as illustrated by gene replacement for X-linked myotubular myopathy (XLMTM) in canine and murine models. Prospective applications and approaches to gene replacement therapy, using ...[more]
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