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Items: 2

1.

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.

Sci Transl Med. 2019 Mar 20;11(484). pii: eaav1866. doi: 10.1126/scitranslmed.aav1866.

PMID:
30894500
2.

Single Intramuscular Injection of AAV-shRNA Reduces DNM2 and Prevents Myotubular Myopathy in Mice.

Tasfaout H, Lionello VM, Kretz C, Koebel P, Messaddeq N, Bitz D, Laporte J, Cowling BS.

Mol Ther. 2018 Apr 4;26(4):1082-1092. doi: 10.1016/j.ymthe.2018.02.008. Epub 2018 Feb 14.

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