Detalhe da pesquisa
1.
Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction.
J Biol Chem
; 298(12): 102625, 2022 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-36306823
2.
Vector genome loss and epigenetic modifications mediate decline in transgene expression of AAV5 vectors produced in mammalian and insect cells.
Mol Ther
; 30(12): 3570-3586, 2022 12 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-36348622
3.
Intermittent enzyme replacement therapy with recombinant human ß-galactosidase prevents neuraminidase 1 deficiency
J Biol Chem
; 295(39): 13556-13569, 2020 09 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-32727849
4.
Intracerebroventricular enzyme replacement therapy with ß-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice.
J Biol Chem
; 295(39): 13532-13555, 2020 09 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-31481471
5.
Quantitative Assessment of Neuroinflammation, Myelinogenesis, Demyelination, and Nerve Fiber Regeneration in Immunostained Sciatic Nerves From Twitcher Mice With a Tissue Image Analysis Platform.
Toxicol Pathol
; 49(4): 950-962, 2021 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33691530
6.
Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice.
Mol Ther
; 26(2): 496-509, 2018 02 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29292164
7.
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.
Proc Natl Acad Sci U S A
; 111(41): 14870-5, 2014 Oct 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-25267636
8.
Correction: Intermittent enzyme replacement therapy prevents Neu1 deficiency.
J Biol Chem
; 295(46): 15794, 2020 Nov 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-33188082
9.
Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia.
Am J Hum Genet
; 91(6): 1108-14, 2012 Dec 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-23200862
10.
Neutral endopeptidase-resistant C-type natriuretic peptide variant represents a new therapeutic approach for treatment of fibroblast growth factor receptor 3-related dwarfism.
J Pharmacol Exp Ther
; 353(1): 132-49, 2015 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-25650377
11.
Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression.
Hum Gene Ther
; 35(1-2): 36-47, 2024 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-38126359
12.
Glycosylphosphatidylinositol-anchored high-density lipoprotein-binding protein 1 plays a critical role in the lipolytic processing of chylomicrons.
Cell Metab
; 5(4): 279-91, 2007 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-17403372
13.
Osteochondroma formation is independent of heparanase expression as revealed in a mouse model of hereditary multiple exostoses.
J Orthop Res
; 40(10): 2391-2401, 2022 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34996123
14.
Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A.
Nat Med
; 28(4): 789-797, 2022 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-35411075
15.
Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers.
Mol Ther Methods Clin Dev
; 24: 142-153, 2022 Mar 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-35036471
16.
Application of in-vitro-cultured primary hepatocytes to evaluate species translatability and AAV transduction mechanisms of action.
Mol Ther Methods Clin Dev
; 26: 61-71, 2022 Sep 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35782594
17.
Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood.
Mol Ther Methods Clin Dev
; 26: 519-531, 2022 Sep 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36092364
18.
Intrathecal tripeptidyl-peptidase 1 reduces lysosomal storage in a canine model of late infantile neuronal ceroid lipofuscinosis.
Mol Genet Metab
; 104(3): 325-37, 2011 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-21784683
19.
Molecular imaging of the initial inflammatory response in atherosclerosis: implications for early detection of disease.
Arterioscler Thromb Vasc Biol
; 30(1): 54-9, 2010 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-19834105
20.
Fish Acquisition and Consumption in the African Great Lakes Region through a Food Environment Lens: A Scoping Review.
Nutrients
; 13(7)2021 Jul 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-34371918