Detalhe da pesquisa
1.
Dual FKRP/FST gene therapy in a single AAV normalizes ambulation, increases muscle strength, decreases muscle pathology, and amplifies gene expression in the FKRPP448L model of LGMDR9.
Mol Ther
; 2024 Jun 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-38910327
2.
Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease.
Mol Ther
; 32(6): 1701-1720, 2024 Jun 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-38549375
3.
Reduced cardiac antioxidant defenses mediate increased susceptibility to workload-induced myocardial injury in males with genetic cardiomyopathy.
J Mol Cell Cardiol
; 190: 24-34, 2024 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-38527667
4.
Osteosarcoma tumors maintain intra-tumoral transcriptional heterogeneity during bone and lung colonization.
BMC Biol
; 21(1): 98, 2023 04 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-37106386
5.
Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies.
Neuropathol Appl Neurobiol
; 48(3): e12785, 2022 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-34847621
6.
Host-derived growth factors drive ERK phosphorylation and MCL1 expression to promote osteosarcoma cell survival during metastatic lung colonization.
Cell Oncol (Dordr)
; 47(1): 259-282, 2024 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-37676378
7.
Single-Stranded DNA with Internal Base Modifications Mediates Highly Efficient Gene Insertion in Primary Cells.
bioRxiv
; 2024 Feb 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-38352420
8.
C9ORF72 patient-derived endothelial cells drive blood-brain barrier disruption and contribute to neurotoxicity.
Fluids Barriers CNS
; 21(1): 34, 2024 Apr 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-38605366
9.
Prothrombin Knockdown Protects Podocytes and Reduces Proteinuria in Glomerular Disease.
bioRxiv
; 2024 Mar 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-38464017
10.
In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans.
Mol Ther Methods Clin Dev
; 30: 16-29, 2023 Sep 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-37746244
11.
Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model.
Mol Ther Methods Clin Dev
; 31: 101144, 2023 Dec 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38027058
12.
Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines.
Front Cell Dev Biol
; 11: 1181040, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37397246
13.
CRISPR-Cas9 homology-independent targeted integration of exons 1-19 restores full-length dystrophin in mice.
Mol Ther Methods Clin Dev
; 30: 486-499, 2023 Sep 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-37706184
14.
Generation and characterization of a human induced pluripotent stem cell (iPSC) line from a patient with congenital heart disease (CHD).
Stem Cell Res
; 65: 102958, 2022 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-36343514
15.
Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse.
Mol Ther Methods Clin Dev
; 26: 279-293, 2022 Sep 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35949298
16.
Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy.
Mol Ther Nucleic Acids
; 30: 479-492, 2022 Dec 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-36420217
17.
A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2.
Mol Ther Methods Clin Dev
; 27: 47-60, 2022 Dec 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36186954
18.
Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping.
Mol Ther Methods Clin Dev
; 21: 325-340, 2021 Jun 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-33898631