Detalhe da pesquisa
1.
Mice lacking MBNL1 and MBNL2 exhibit sudden cardiac death and molecular signatures recapitulating myotonic dystrophy.
Hum Mol Genet
; 31(18): 3144-3160, 2022 09 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-35567413
2.
Repeat length increases disease penetrance and severity in C9orf72 ALS/FTD BAC transgenic mice.
Hum Mol Genet
; 29(24): 3900-3918, 2021 02 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-33378537
3.
Precise small-molecule cleavage of an r(CUG) repeat expansion in a myotonic dystrophy mouse model.
Proc Natl Acad Sci U S A
; 116(16): 7799-7804, 2019 04 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-30926669
4.
Transcriptome alterations in myotonic dystrophy skeletal muscle and heart.
Hum Mol Genet
; 28(8): 1312-1321, 2019 04 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-30561649
5.
Transcriptome-Wide Studies of RNA-Targeted Small Molecules Provide a Simple and Selective r(CUG)exp Degrader in Myotonic Dystrophy.
ACS Cent Sci
; 9(7): 1342-1353, 2023 Jul 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-37521782
6.
The skeletal muscle circadian clock regulates titin splicing through RBM20.
Elife
; 112022 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36047761
7.
High-content image-based analysis and proteomic profiling identifies Tau phosphorylation inhibitors in a human iPSC-derived glutamatergic neuronal model of tauopathy.
Sci Rep
; 11(1): 17029, 2021 08 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-34426604
8.
Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G4C2) repeat expansion in vitro and in vivo ALS models.
Sci Transl Med
; 13(617): eabd5991, 2021 Oct 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-34705518