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1.
Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy.
Gene Ther
; 29(10-11): 608-615, 2022 11.
Article
in English
| MEDLINE | ID: mdl-34737451
2.
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model.
Gene Ther
; 29(9): 520-535, 2022 Sep.
Article
in English
| MEDLINE | ID: mdl-35105949
3.
TRPC3, but not TRPC1, as a good therapeutic target for standalone or complementary treatment of DMD.
J Transl Med
; 19(1): 519, 2021 12 20.
Article
in English
| MEDLINE | ID: mdl-34930315
4.
Dystrophic Dmdmdx rats show early neuronal changes (increased S100ß and Tau5) at 8 months, supporting severe dystropathology in this rodent model of Duchenne muscular dystrophy.
Mol Cell Neurosci
; 108: 103549, 2020 10.
Article
in English
| MEDLINE | ID: mdl-32890728
5.
Gait characterization in golden retriever muscular dystrophy dogs using linear discriminant analysis.
BMC Musculoskelet Disord
; 18(1): 153, 2017 04 12.
Article
in English
| MEDLINE | ID: mdl-28403854
6.
Serum profiling identifies novel muscle miRNA and cardiomyopathy-related miRNA biomarkers in Golden Retriever muscular dystrophy dogs and Duchenne muscular dystrophy patients.
Am J Pathol
; 184(11): 2885-98, 2014 Nov.
Article
in English
| MEDLINE | ID: mdl-25194663
7.
Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates.
Mol Ther
; 22(9): 1605-13, 2014 Sep.
Article
in English
| MEDLINE | ID: mdl-24954473
8.
Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.
Mol Ther
; 22(11): 1923-35, 2014 Nov.
Article
in English
| MEDLINE | ID: mdl-25200009
9.
Dystrophin deficiency impairs cell junction formation during embryonic myogenesis.
bioRxiv
; 2024 Apr 08.
Article
in English
| MEDLINE | ID: mdl-38106055
10.
Novel chemical tyrosine functionalization of adeno-associated virus improves gene transfer efficiency in liver and retina.
Biomed Pharmacother
; 171: 116148, 2024 Feb.
Article
in English
| MEDLINE | ID: mdl-38232661
11.
Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency.
Mol Ther Methods Clin Dev
; 32(1): 101187, 2024 Mar 14.
Article
in English
| MEDLINE | ID: mdl-38327809
12.
PCR-based detection of gene transfer vectors: application to gene doping surveillance.
Anal Bioanal Chem
; 405(30): 9641-53, 2013 Dec.
Article
in English
| MEDLINE | ID: mdl-23912835
13.
Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver.
Mol Ther
; 20(6): 1177-86, 2012 Jun.
Article
in English
| MEDLINE | ID: mdl-22453768
14.
Muscle Pathology in Dystrophic Rats and Zebrafish Is Unresponsive to Taurine Treatment, Compared to the mdx Mouse Model for Duchenne Muscular Dystrophy.
Metabolites
; 13(2)2023 Feb 04.
Article
in English
| MEDLINE | ID: mdl-36837851
15.
Intravitreal air tamponade after AAV2 subretinal injection modifies retinal EGFP distribution.
Mol Ther Methods Clin Dev
; 28: 387-393, 2023 Mar 09.
Article
in English
| MEDLINE | ID: mdl-36874242
16.
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy.
Mol Ther Methods Clin Dev
; 30: 30-47, 2023 Sep 14.
Article
in English
| MEDLINE | ID: mdl-37746247
17.
Systemic delivery of allogenic muscle stem cells induces long-term muscle repair and clinical efficacy in duchenne muscular dystrophy dogs.
Am J Pathol
; 179(5): 2501-18, 2011 Nov.
Article
in English
| MEDLINE | ID: mdl-21924229
18.
AAV2/9-mediated gene transfer into murine lacrimal gland leads to a long-term targeted tear film modification.
Mol Ther Methods Clin Dev
; 27: 1-16, 2022 Dec 08.
Article
in English
| MEDLINE | ID: mdl-36156877
19.
Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle.
Mol Ther
; 18(1): 151-60, 2010 Jan.
Article
in English
| MEDLINE | ID: mdl-19888197
20.
AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation.
Mol Ther Methods Clin Dev
; 20: 660-674, 2021 Mar 12.
Article
in English
| MEDLINE | ID: mdl-33718516