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Short Communication |
Infectious delivery and long-term persistence of transgene expression in the brain by a 135-kb iBAC-FXN genomic DNA expression vector
- A Gimenez-Cassina
- , R Wade-Martins
- & J Diaz-Nido
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Original Article
| Open AccessAAV-mediated in vivo knockdown of luciferase using combinatorial RNAi and U1i
- A Koornneef
- , R van Logtenstein
- & T Ritsema
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Original Article |
Hematopoietic gene therapy restores thymidine phosphorylase activity in a cell culture and a murine model of MNGIE
- J Torres-Torronteras
- , A Gómez
- & R Martí
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Original Article |
Improvement of cardiac fibrosis in dystrophic mice by rAAV9-mediated microdystrophin transduction
- J-H Shin
- , Y Nitahara-Kasahara
- & S Takeda
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Original Article |
Long-term phenotypic correction in factor IX knockout mice by using phiC31 integrase-mediated gene therapy
- A Keravala
- , C L Chavez
- & M P Calos
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Original Article
| Open AccessAAV-mediated photoreceptor transduction of the pig cone-enriched retina
- C Mussolino
- , M della Corte
- & E M Surace
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Original Article |
Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping
- W Ni
- , C Le Guiner
- & R O Snyder
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Short Communication |
In vitro and in vivo comparison of viral and cellular internal ribosome entry sites for bicistronic vector expression
- M Licursi
- , S L Christian
- & K Hirasawa
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Original Article |
Lethal toxicity caused by expression of shRNA in the mouse striatum: implications for therapeutic design
- J N Martin
- , N Wolken
- & P Gonzalez-Alegre
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Original Article |
MMP-2 mediates mesenchymal stem cell tropism towards medulloblastoma tumors
- P Bhoopathi
- , C Chetty
- & S S Lakka
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Original Article |
Highly efficient multipotent differentiation of human periodontal ligament fibroblasts induced by combined BMP4 and hTERT gene transfer
- H-W Mi
- , M-C Lee
- & C-P Lin
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Original Article |
Overexpression of X-linked inhibitor of apoptosis protein protects against noise-induced hearing loss in mice
- J Wang
- , N Tymczyszyn
- & G S Robertson
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Short Communication |
APOBEC3-mediated hypermutation of retroviral vectors produced from some retrovirus packaging cell lines
- A D Miller
- & M J Metzger
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Original Article |
Adeno-associated virus-mediated gene delivery into the scala media of the normal and deafened adult mouse ear
- L A Kilpatrick
- , Q Li
- & H Lang
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Original Article |
Evaluation of delivery conditions for cutaneous plasmid electrotransfer using a multielectrode array
- B Ferraro
- , L C Heller
- & R Heller
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Enabling Technologies
| Open AccessDown-modulation of cancer targets using locked nucleic acid (LNA)-based antisense oligonucleotides without transfection
- Y Zhang
- , Z Qu
- & I D Horak
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Original Article |
Construction of a novel expression cassette for increasing transgene expression in vivo in endothelial cells of large blood vessels
- N Dronadula
- , L Du
- & D A Dichek
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Original Article |
Matrix metalloproteinase 14 overexpression reduces corneal scarring
- S D Galiacy
- , P Fournié
- & F Malecaze
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Original Article |
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver
- C Qiao
- , Z Yuan
- & X Xiao
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Original Article |
A tissue-specific, activation-inducible, lentiviral vector regulated by human CD40L proximal promoter sequences
- Z Romero
- , S Torres
- & I J Molina
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Enabling Technologies |
Improved antibiotic-free plasmid vector design by incorporation of transient expression enhancers
- J M Luke
- , J M Vincent
- & J A Williams
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Original Article |
Transient expression of OCT4 is sufficient to allow human keratinocytes to change their differentiation pathway
- D Racila
- , M Winter
- & J R Bickenbach
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Review |
Physiological and tissue-specific vectors for treatment of inherited diseases
- M G Toscano
- , Z Romero
- & F Martin
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Original Article |
Minimal doses of a sequence-optimized transgene mediate high-level and long-term EPO expression in vivo: challenging CpG-free gene design
- D Kosovac
- , J Wild
- & R Wagner
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Original Article |
Effects of irradiating adult mdx mice before full-length dystrophin cDNA transfer on host anti-dystrophin immunity
- S Eghtesad
- , H Zheng
- & P R Clemens
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Enabling Technologies |
Use of human MAR elements to improve retroviral vector production
- M Buceta
- , J L Galbete
- & N Mermod
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Original Article |
Metabolically stabilized long-circulating PEGylated polyacridine peptide polyplexes mediate hydrodynamically stimulated gene expression in liver
- C A Fernandez
- , N J Baumhover
- & K G Rice
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Original Article |
Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution
- K-M R Prasad
- , Y Xu
- & B A French
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Original Article |
Second-generation HIF-activated oncolytic adenoviruses with improved replication, oncolytic, and antitumor efficacy
- T Cherry
- , S L Longo
- & D E Post
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Original Article |
CFTR expression and activity from the human CFTR locus in BAC vectors, with regulatory regions, isolated by a single-step procedure
- C Auriche
- , E G Di Domenico
- & F Ascenzioni
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Original Article |
Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector
- P S Becker
- , J A Taylor
- & H-P Kiem
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Original Article |
Gene doping detection: evaluation of approach for direct detection of gene transfer using erythropoietin as a model system
- A Baoutina
- , T Coldham
- & K R Emslie
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Enabling Technologies |
Signed outside: a surface marker system for transgenic cytoplasmic proteins
- V Wohlgensinger
- , R Seger
- & U Siler
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Original Article |
Noninvasive assessment of regulable transferred-p53 gene expression and evaluation of therapeutic response with FDG–PET in tumor model
- W Aung
- , S Hasegawa
- & T Saga
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Original Article |
Retargeted adenoviral cancer gene therapy for tumour cells overexpressing epidermal growth factor receptor or urokinase-type plasminogen activator receptor
- T J Harvey
- , D Burdon
- & J D Chester
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Original Article |
Duration and level of transgene expression after gene electrotransfer to skin in mice
- A Gothelf
- , J Eriksen
- & J Gehl
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Short Communication |
Immune reactivity to type VII collagen: implications for gene therapy of recessive dystrophic epidermolysis bullosa
- V Pendaries
- , G Gasc
- & A Hovnanian
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Original Article |
Generation of human TRIM5α mutants with high HIV-1 restriction activity
- Q T Pham
- , A Bouchard
- & L Berthoux
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Original Article |
Anticonvulsant effects and behavioural outcomes of rAAV serotype 1 vector-mediated neuropeptide Y overexpression in rat hippocampus
- F Noe
- , V Vaghi
- & A Vezzani
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Original Article |
Organ-specific shifts in mtDNA heteroplasmy following systemic delivery of a mitochondria-targeted restriction endonuclease
- S R Bacman
- , S L Williams
- & C T Moraes
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Original Article |
Functional recovery of diabetic mouse hearts by glutaredoxin-1 gene therapy: role of Akt-FoxO-signaling network
- I Lekli
- , S Mukherjee
- & D K Das
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Original Article |
Rhadinovirus vector-derived human telomerase reverse transcriptase expression in primary T cells
- T Toptan
- , A Ensser
- & H Fickenscher
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News and Commentary |
The battle to prevent the transmission of mitochondrial DNA disease: Is karyoplast transfer the answer?
- J C St John
- & K H S Campbell
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Original Article |
The SP-C promoter facilitates alveolar type II epithelial cell-specific plasmid nuclear import and gene expression
- J V DeGiulio
- , C D Kaufman
- & D A Dean
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