Detalhe da pesquisa
1.
Vector integration and fate in the hemophilia dog liver multiple years after AAV-FVIII gene transfer.
Blood
; 143(23): 2373-2385, 2024 Jun 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-38452208
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.
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
4.
Etiology of distinct membrane excitability in pre- and posthearing auditory neurons relies on activity of Cl- channel TMEM16A.
Proc Natl Acad Sci U S A
; 112(8): 2575-80, 2015 Feb 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-25675481
5.
Mechanisms of Calmodulin Regulation of Different Isoforms of Kv7.4 K+ Channels.
J Biol Chem
; 291(5): 2499-509, 2016 Jan 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-26515070
6.
Genetic, cellular, and functional evidence for Ca2+ inflow through Cav1.2 and Cav1.3 channels in murine spiral ganglion neurons.
J Neurosci
; 34(21): 7383-93, 2014 May 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-24849370
7.
Functional significance of K+ channel ß-subunit KCNE3 in auditory neurons.
J Biol Chem
; 289(24): 16802-13, 2014 Jun 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-24727472
8.
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
9.
The Piezo channel is a mechano-sensitive complex component in the mammalian inner ear hair cell.
Nat Commun
; 15(1): 526, 2024 Jan 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-38228630
10.
Posthearing Ca(2+) currents and their roles in shaping the different modes of firing of spiral ganglion neurons.
J Neurosci
; 32(46): 16314-30, 2012 Nov 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-23152615
11.
The Piezo channel is central to the mechano-sensitive channel complex in the mammalian inner ear.
Res Sq
; 2023 Jul 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-37502846
12.
Cellular and molecular mechanisms of autosomal dominant form of progressive hearing loss, DFNA2.
J Biol Chem
; 286(2): 1517-27, 2011 Jan 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-20966080
13.
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
14.
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
15.
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
16.
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
17.
Prestin amplifies cardiac motor functions.
Cell Rep
; 35(5): 109097, 2021 05 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33951436
18.
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
19.
Different arrhythmia-associated calmodulin mutations have distinct effects on cardiac SK channel regulation.
J Gen Physiol
; 152(12)2020 12 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-33211795
20.
Cooperativity of Kv7.4 channels confers ultrafast electromechanical sensitivity and emergent properties in cochlear outer hair cells.
Sci Adv
; 6(15): eaba1104, 2020 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-32285007