Detalhe da pesquisa
1.
Obesity-associated gene TMEM18 has a role in the central control of appetite and body weight regulation.
Proc Natl Acad Sci U S A
; 114(35): 9421-9426, 2017 08 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-28811369
2.
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
3.
Relevance of Assembly-Activating Protein for Adeno-associated Virus Vector Production and Capsid Protein Stability in Mammalian and Insect Cells.
J Virol
; 91(20)2017 10 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-28768875
4.
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
5.
Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency.
Mol Ther Methods Clin Dev
; 32(1): 101187, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38327809
6.
Novel chemical tyrosine functionalization of adeno-associated virus improves gene transfer efficiency in liver and retina.
Biomed Pharmacother
; 171: 116148, 2024 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-38232661
7.
Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice.
Mol Ther
; 20(2): 254-66, 2012 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-22008915
8.
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
9.
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates.
Nat Commun
; 13(1): 2454, 2022 05 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-35508619
10.
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells.
Cell Stem Cell
; 29(10): 1428-1444.e9, 2022 10 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36206730
11.
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
12.
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
13.
Baboon Envelope Pseudotyped "Nanoblades" Carrying Cas9/gRNA Complexes Allow Efficient Genome Editing in Human T, B, and CD34+ Cells and Knock-in of AAV6-Encoded Donor DNA in CD34+ Cells.
Front Genome Ed
; 3: 604371, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34713246
14.
Increased intraocular insulin-like growth factor-I triggers blood-retinal barrier breakdown.
J Biol Chem
; 284(34): 22961-9, 2009 Aug 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-19473988
15.
Endothelial cell transduction in primary cultures from regressing mesonephros.
Cells Tissues Organs
; 191(2): 84-95, 2010.
Artigo
em Inglês
| MEDLINE | ID: mdl-19641291
16.
Intrinsic Differential Scanning Fluorimetry for Fast and Easy Identification of Adeno-Associated Virus Serotypes.
J Pharm Sci
; 109(1): 854-862, 2020 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31639391
17.
Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries.
Mol Ther Methods Clin Dev
; 17: 107-121, 2020 Jun 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-31909084
18.
Single-Stranded DNA Virus Sequencing (SSV-Seq) for Characterization of Residual DNA and AAV Vector Genomes.
Methods Mol Biol
; 1950: 85-106, 2019.
Artigo
em Inglês
| MEDLINE | ID: mdl-30783969
19.
Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates.
Sci Transl Med
; 11(493)2019 05 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-31118293
20.
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