Detalhe da pesquisa
1.
AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.
Mol Ther
; 27(9): 1568-1585, 2019 09 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-31327755
2.
Nitric oxide-dependent attenuation of noradrenaline-induced vasoconstriction is impaired in the canine model of Duchenne muscular dystrophy.
J Physiol
; 596(21): 5199-5216, 2018 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-30152022
3.
Extensor carpi ulnaris muscle shows unexpected slow-to-fast fiber-type switch in Duchenne muscular dystrophy dogs.
Dis Model Mech
; 14(12)2021 12 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34704592
4.
Widespread severe myodegeneration in a compound heterozygote female dog with dystrophin deficiency.
Vet Med Sci
; 7(3): 654-659, 2021 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33502125
5.
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.
Nat Commun
; 12(1): 6769, 2021 11 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-34819506
6.
High prevalence of plasma lipid abnormalities in human and canine Duchenne and Becker muscular dystrophies depicts a new type of primary genetic dyslipidemia.
J Clin Lipidol
; 14(4): 459-469.e0, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-32593511
7.
Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease.
Mol Ther Methods Clin Dev
; 18: 664-678, 2020 Sep 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-32775499
8.
Systemic Delivery of Adeno-Associated Viral Vectors in Mice and Dogs.
Methods Mol Biol
; 1937: 281-294, 2019.
Artigo
em Inglês
| MEDLINE | ID: mdl-30706404
9.
Questions Answered and Unanswered by the First CRISPR Editing Study in a Canine Model of Duchenne Muscular Dystrophy.
Hum Gene Ther
; 30(5): 535-543, 2019 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-30648435
10.
Nanotherapy for Duchenne muscular dystrophy.
Wiley Interdiscip Rev Nanomed Nanobiotechnol
; 10(2)2018 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28398005
11.
Automatic characterization of stride parameters in canines with a single wearable inertial sensor.
PLoS One
; 13(6): e0198893, 2018.
Artigo
em Inglês
| MEDLINE | ID: mdl-29902280
12.
Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model.
Hum Gene Ther
; 29(3): 299-311, 2018 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28793798
13.
Repurposing a novel parathyroid hormone analogue to treat hypoparathyroidism.
Br J Pharmacol
; 175(2): 262-271, 2018 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28898923
14.
AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice.
JCI Insight
; 3(23)2018 12 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-30518686
15.
A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy.
Mol Ther Methods Clin Dev
; 6: 216-230, 2017 Sep 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-28932757
16.
Financing translation: analysis of the NCATS rare-diseases portfolio.
Sci Transl Med
; 7(276): 276ps3, 2015 Feb 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-25717096
17.
Gene Therapy: The View from NCATS.
Hum Gene Ther
; 27(1): 7-13, 2016 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-26784641