Detalhe da pesquisa
1.
Elimination of CaMKIIδ Autophosphorylation by CRISPR-Cas9 Base Editing Improves Survival and Cardiac Function in Heart Failure in Mice.
Circulation
; 148(19): 1490-1504, 2023 11 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-37712250
2.
Partial F8 gene duplication (factor VIII Padua) associated with high factor VIII levels and familial thrombophilia.
Blood
; 137(17): 2383-2393, 2021 04 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-33275657
3.
Degenerative and regenerative pathways underlying Duchenne muscular dystrophy revealed by single-nucleus RNA sequencing.
Proc Natl Acad Sci U S A
; 117(47): 29691-29701, 2020 11 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-33148801
4.
Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing.
Mol Ther
; 28(9): 2044-2055, 2020 09 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-32892813
5.
Single cell analysis reveals the involvement of the long non-coding RNA Pvt1 in the modulation of muscle atrophy and mitochondrial network.
Nucleic Acids Res
; 47(4): 1653-1670, 2019 02 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-30649422
6.
A Single Cell but Many Different Transcripts: A Journey into the World of Long Non-Coding RNAs.
Int J Mol Sci
; 21(1)2020 Jan 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31906285
7.
Involvement of microRNAs in the regulation of muscle wasting during catabolic conditions.
J Biol Chem
; 289(32): 21909-25, 2014 Aug 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-24891504
8.
Synthesis of mitochondrial DNA precursors during myogenesis, an analysis in purified C2C12 myotubes.
J Biol Chem
; 288(8): 5624-35, 2013 Feb 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-23297407
9.
CRISPR-Editing Therapy for Duchenne Muscular Dystrophy.
Hum Gene Ther
; 34(9-10): 379-387, 2023 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-37060194
10.
Dysfunctional mitochondria accumulate in a skeletal muscle knockout model of Smn1, the causal gene of spinal muscular atrophy.
Cell Death Dis
; 14(2): 162, 2023 02 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-36849544
11.
Ablation of CaMKIIδ oxidation by CRISPR-Cas9 base editing as a therapy for cardiac disease.
Science
; 379(6628): 179-185, 2023 01 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-36634166
12.
Base editing correction of hypertrophic cardiomyopathy in human cardiomyocytes and humanized mice.
Nat Med
; 29(2): 401-411, 2023 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36797478
13.
Net39 protects muscle nuclei from mechanical stress during the pathogenesis of Emery-Dreifuss muscular dystrophy.
J Clin Invest
; 133(13)2023 07 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-37395273
14.
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations.
Mol Ther Nucleic Acids
; 32: 522-535, 2023 Jun 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-37215149
15.
Long-term maintenance of dystrophin expression and resistance to injury of skeletal muscle in gene edited DMD mice.
Mol Ther Nucleic Acids
; 28: 154-167, 2022 Jun 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-35402069
16.
Impaired activity of the fusogenic micropeptide Myomixer causes myopathy resembling Carey-Fineman-Ziter syndrome.
J Clin Invest
; 132(11)2022 06 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35642635
17.
MyoData: An expression knowledgebase at single cell/nucleus level for the discovery of coding-noncoding RNA functional interactions in skeletal muscle.
Comput Struct Biotechnol J
; 19: 4142-4155, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34527188
18.
Correction of muscular dystrophies by CRISPR gene editing.
J Clin Invest
; 130(6): 2766-2776, 2020 06 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32478678
19.
Isolation and Transcriptomic Profiling of Single Myofibers from Mice.
Bio Protoc
; 9(19): e3378, 2019 Oct 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33654874
20.
Helicobacter pylori Dampens HLA-II Expression on Macrophages via the Up-Regulation of miRNAs Targeting CIITA.
Front Immunol
; 10: 2923, 2019.
Artigo
em Inglês
| MEDLINE | ID: mdl-31969878