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Multi-omics comparisons of different forms of centronuclear myopathies and the effects of several therapeutic strategies.
Mol Ther. 2021 Aug 4;29(8):2514-2534. doi: 10.1016/j.ymthe.2021.04.033. Epub 2021 May 1.
Mol Ther. 2021.
PMID: 33940157
Free PMC article.
Amphiphysin 2 modulation rescues myotubular myopathy and prevents focal adhesion defects in mice.
Lionello VM, Nicot AS, Sartori M, Kretz C, Kessler P, Buono S, Djerroud S, Messaddeq N, Koebel P, Prokic I, Hérault Y, Romero NB, Laporte J, Cowling BS.
Lionello VM, et al. Among authors: djerroud s.
Sci Transl Med. 2019 Mar 20;11(484):eaav1866. doi: 10.1126/scitranslmed.aav1866.
Sci Transl Med. 2019.
PMID: 30894500
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