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.
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
6.
Correction: Intermittent enzyme replacement therapy prevents Neu1 deficiency.
J Biol Chem
; 295(46): 15794, 2020 Nov 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-33188082
7.
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
8.
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
9.
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
10.
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
11.
Prednisolone Does Not Regulate Factor VIII Expression in Mice Receiving AAV5-hFVIII-SQ: Valoctocogene Roxaparvovec.
Mol Ther Methods Clin Dev
; 17: 13-20, 2020 Jun 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-31890737
12.
Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter.
Mol Ther Methods Clin Dev
; 18: 620-630, 2020 Sep 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-32775496