Detalhe da pesquisa
1.
Removal of the GAA repeat in the heart of a Friedreich's ataxia mouse model using CjCas9.
Gene Ther
; 30(7-8): 612-619, 2023 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36781946
2.
Serum extracellular vesicles for delivery of CRISPR-CAS9 ribonucleoproteins to modify the dystrophin gene.
Mol Ther
; 30(7): 2429-2442, 2022 07 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35619556
3.
CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing.
Gene Ther
; 29(3-4): 171-177, 2022 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-34593991
4.
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mol Ther
; 29(2): 464-488, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33309881
5.
Prime Editing Permits the Introduction of Specific Mutations in the Gene Responsible for Duchenne Muscular Dystrophy.
Int J Mol Sci
; 23(11)2022 May 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-35682838
6.
CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo.
Mol Ther
; 26(11): 2604-2616, 2018 11 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-30195724
7.
Nonfunctional mutant Wrn protein leads to neurological deficits, neuronal stress, microglial alteration, and immune imbalance in a mouse model of Werner syndrome.
Brain Behav Immun
; 73: 450-469, 2018 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-29908963
8.
Three Decades of Clinical Gene Therapy: From Experimental Technologies to Viable Treatments.
Mol Ther
; 29(2): 411-412, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33472032
9.
Generation of human endometrial knockout cell lines with the CRISPR/Cas9 system confirms the prostaglandin F2α synthase activity of aldo-ketoreductase 1B1.
Mol Hum Reprod
; 20(7): 650-63, 2014 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-24674991
10.
Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients.
Mol Ther
; 21(9): 1718-26, 2013 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-23732986
11.
Therapeutic effects of exon skipping and losartan on skeletal muscle of mdx mice.
Pathol Int
; 64(8): 388-96, 2014 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-25143127
12.
Human MuStem cells are competent to fuse with nonhuman primate myofibers in a clinically relevant transplantation context: A proof-of-concept study.
J Neuropathol Exp Neurol
; 2024 May 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-38752570
13.
Myoblasts derived from normal hESCs and dystrophic hiPSCs efficiently fuse with existing muscle fibers following transplantation.
Mol Ther
; 20(11): 2153-67, 2012 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-22990676
14.
Prime Editing for Human Gene Therapy: Where Are We Now?
Cells
; 12(4)2023 02 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-36831203
15.
Limb-Girdle Muscular Dystrophies Classification and Therapies.
J Clin Med
; 12(14)2023 Jul 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-37510884
16.
Gene therapy for Duchenne muscular dystrophy: an update on the latest clinical developments.
Expert Rev Neurother
; 23(10): 905-920, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37602688
17.
Portrait of Dysferlinopathy: Diagnosis and Development of Therapy.
J Clin Med
; 12(18)2023 Sep 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-37762951
18.
CRISPR-Cas9 delivery strategies with engineered extracellular vesicles.
Mol Ther Nucleic Acids
; 34: 102040, 2023 Dec 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-37842166
19.
Successful Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathy.
Cells
; 13(1)2023 12 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-38201236
20.
A promising mouse model for Friedreich Ataxia progressing like human patients.
Behav Brain Res
; 436: 114107, 2023 01 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-36089099