Detalhe da pesquisa
1.
Analytical ultracentrifugation sedimentation velocity for the characterization of recombinant adeno-associated virus vectors sub-populations.
Eur Biophys J
; 52(4-5): 367-377, 2023 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-37106255
2.
Correction of a knock-in mouse model of acrodysostosis with gene therapy using a rAAV9-CAG-human PRKAR1A vector.
Gene Ther
; 29(7-8): 441-448, 2022 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-34599290
3.
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.
Artigo
em Inglês
| MEDLINE | ID: mdl-35105949
4.
Stability of the adeno-associated virus 8 reference standard material.
Gene Ther
; 26(5): 211-215, 2019 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-30926961
5.
AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations.
Mol Ther
; 25(6): 1375-1386, 2017 06 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-28427840
6.
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.
Artigo
em Inglês
| MEDLINE | ID: mdl-24954473
7.
MyD88 signaling in B cells regulates the production of Th1-dependent antibodies to AAV.
Mol Ther
; 20(8): 1571-81, 2012 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-22643865
8.
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.
Artigo
em Inglês
| MEDLINE | ID: mdl-36156877
9.
Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity.
Hum Gene Ther
; 33(7-8): 371-388, 2022 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-35293222
10.
The SSV-Seq 2.0 PCR-Free Method Improves the Sequencing of Adeno-Associated Viral Vector Genomes Containing GC-Rich Regions and Homopolymers.
Biotechnol J
; 16(1): e2000016, 2021 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-33064875
11.
Homologous Recombination Offers Advantages over Transposition-Based Systems to Generate Recombinant Baculovirus for Adeno-Associated Viral Vector Production.
Biotechnol J
; 16(1): e2000014, 2021 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-33067902
12.
Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
Mol Ther Methods Clin Dev
; 17: 771-784, 2020 Jun 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-32355866
13.
Chemical modification of the adeno-associated virus capsid to improve gene delivery.
Chem Sci
; 11(4): 1122-1131, 2019 Dec 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-34084369
14.
The role of the adeno-associated virus capsid in gene transfer.
Methods Mol Biol
; 437: 51-91, 2008.
Artigo
em Inglês
| MEDLINE | ID: mdl-18369962
15.
Accurate Titration of Infectious AAV Particles Requires Measurement of Biologically Active Vector Genomes and Suitable Controls.
Mol Ther Methods Clin Dev
; 10: 223-236, 2018 Sep 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-30112419
16.
Accurate Identification and Quantification of DNA Species by Next-Generation Sequencing in Adeno-Associated Viral Vectors Produced in Insect Cells.
Hum Gene Ther Methods
; 28(3): 148-162, 2017 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-28463571
17.
A fragmented adeno-associated viral dual vector strategy for treatment of diseases caused by mutations in large genes leads to expression of hybrid transcripts.
J Genet Syndr Gene Ther
; 7(5)2016 Nov 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-28239514
18.
Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR.
Mol Ther Methods Clin Dev
; 5: 16019, 2016.
Artigo
em Inglês
| MEDLINE | ID: mdl-27069952
19.
Vitrectomy Before Intravitreal Injection of AAV2/2 Vector Promotes Efficient Transduction of Retinal Ganglion Cells in Dogs and Nonhuman Primates.
Hum Gene Ther Methods
; 27(3): 122-34, 2016 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-27229628
20.
Use of Adeno-Associated Virus to Enrich Cardiomyocytes Derived from Human Stem Cells.
Hum Gene Ther Clin Dev
; 26(3): 194-201, 2015 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-26252064