Detalhe da pesquisa
1.
Mice with muscle-specific deletion of Bin1 recapitulate centronuclear myopathy and acute downregulation of dynamin 2 improves their phenotypes.
Mol Ther
; 30(2): 868-880, 2022 02 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-34371181
2.
Single Intramuscular Injection of AAV-shRNA Reduces DNM2 and Prevents Myotubular Myopathy in Mice.
Mol Ther
; 26(4): 1082-1092, 2018 04 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-29506908
3.
Generation of an MTM1-mutant iPSC line (CRICKi008-A) from an individual with X-linked myotubular myopathy (XLMTM).
Stem Cell Res
; 69: 103079, 2023 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36989620
4.
3D human induced pluripotent stem cell-derived bioengineered skeletal muscles for tissue, disease and therapy modeling.
Nat Protoc
; 18(4): 1337-1376, 2023 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-36792780
5.
Amphiphysin 2 modulation rescues myotubular myopathy and prevents focal adhesion defects in mice.
Sci Transl Med
; 11(484)2019 03 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-30894500