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Original Article |
Recombinant elastin-based nanoparticles for targeted gene therapy
- D A Monfort
- & P Koria
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Comment |
Will rising prices and budget constraints prevent patients from accessing novel gene therapies?
- A Kent
- & J Spink
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Original Article |
Targeting the CXCR4/CXCL12 axis in treating epithelial ovarian cancer
- T L Mao
- , K F Fan
- & C L Liu
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Original Article |
A new combination therapy for asthma using dual-function dexamethasone-conjugated polyethylenimine and vitamin D binding protein siRNA
- M Choi
- , J Gu
- & T Rhim
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Original Article |
Bifidobacterium breve as a delivery vector of IL-24 gene therapy for head and neck squamous cell carcinoma in vivo
- L Wang
- , I Vuletic
- & C Guo
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Original Article
| Open Access
AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease
- J Miniarikova
- , V Zimmer
- & P Konstantinova
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Original Article |
Systemic gene delivery transduces the enteric nervous system of guinea pigs and cynomolgus macaques
- S E Gombash
- , C J Cowley
- & K D Foust
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Review |
Management of neuromuscular diseases and spinal muscular atrophy in Latin America
- S Monges
- & A L Rosa
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Review
| Open Access
The importance of international collaboration for rare diseases research: a European perspective
- D Julkowska
- , C P Austin
- & S van Weely
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Review |
The SMA Trust: the role of a disease-focused research charity in developing treatments for SMA
- V Christie-Brown
- , J Mitchell
- & K Talbot
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Original Article |
INSR gene polymorphisms correlate with sensitivity to platinum-based chemotherapy and prognosis in patients with epithelial ovarian cancer
- J-L Hu
- , X-L Hu
- & S-D Cang
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Review |
Genetic pharmacology: progresses in siRNA delivery and therapeutic applications
- D Scherman
- , A Rousseau
- & V Escriou
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Original Article |
Local gene therapy with indoleamine 2,3-dioxygenase protects against development of transplant vasculopathy in chronic kidney transplant dysfunction
- D Vavrincova-Yaghi
- , L E Deelman
- & M Sandovici
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Original Article |
Exposure to hypomethylating agent, 5-azacytidine, may improve iCasp9 suicide gene therapy for treating GvHD in allografts
- E Bôle-Richard
- , C Gamonet
- & M Deschamps
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Original Article
| Open Access
Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice
- C D Anderson
- , S Moisyadi
- & R V Shohet
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Original Article |
Adenoviral vector encoding soluble Flt-1 engineered human endometrial mesenchymal stem cells effectively regress endometriotic lesions in NOD/SCID mice
- A R Koippallil Gopalakrishnan
- , H Pandit
- & T Madan
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Original Article |
Hair cell stereociliary bundle regeneration by espin gene transduction after aminoglycoside damage and hair cell induction by Notch inhibition
- A Taura
- , K Taura
- & A F Ryan
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Original Article - Enabling Technologies
| Open Access
A new plasmid-based microRNA inhibitor system that inhibits microRNA families in transgenic mice and cells: a potential new therapeutic reagent
- H Cao
- , W Yu
- & B A Amendt
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Original Article |
Treatment of colon cancer with oncolytic herpes simplex virus in preclinical models
- H Yang
- , T Peng
- & B Liu
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Original Article |
Mifepristone-inducible transgene expression in neural progenitor cells in vitro and in vivo
- B E Hjelm
- , C Grunseich
- & T M Pierson
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Original Article
| Open Access
Overexpression of a glucokinase point mutant in the treatment of diabetes mellitus
- G Lu
- , X Teng
- & H Xiong
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Original Article |
Co-expression of fibulin-5 and VEGF165 increases long-term patency of synthetic vascular grafts seeded with autologous endothelial cells
- M Preis
- , J Schneiderman
- & M Y Flugelman
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Original Article |
Gene transfer of two entry inhibitors protects CD4+ T cell from HIV-1 infection in humanized mice
- N Y Petit
- , C Baillou
- & G Marodon
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Original Article |
Evaluation of helper-dependent canine adenovirus vectors in a 3D human CNS model
- D Simão
- , C Pinto
- & P M Alves
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Original Article |
BTK gene targeting by homologous recombination using a helper-dependent adenovirus/adeno-associated virus hybrid vector
- H Yamamoto
- , M Ishimura
- & T Hara
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Original Article |
Helper-dependent adenovirus achieve more efficient and persistent liver transgene expression in non-human primates under immunosuppression
- C Unzu
- , I Melero
- & A Fontanellas
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Original Article |
In vitro screening of clinical drugs identifies sensitizers of oncolytic viral therapy in glioblastoma stem-like cells
- L M E Berghauser Pont
- , R K Balvers
- & M L M Lamfers
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Original Article |
Inhalation of recombinant adenovirus expressing granulysin protects mice infected with Mycobacterium tuberculosis
- J Ma
- , J Lu
- & X Fan
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Original Article |
Tightly regulated ‘all-in-one’ lentiviral vectors for protection of human hematopoietic cells from anticancer chemotherapy
- N Lachmann
- , S Brennig
- & T Moritz
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Original Article |
Neutralizing circulating ghrelin by expressing a growth hormone secretagogue receptor-based protein protects against high-fat diet-induced obesity in mice
- J Gagnon
- , L Zhu
- & Q Wang
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Original Article |
Local administration of AAV-DJ pseudoserotype expressing COX2 provided early onset of transgene expression and promoted bone fracture healing in mice
- R Lakhan
- , D J Baylink
- & X Qin
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Original Article |
Ultrasound-assisted nonviral gene transfer of AQP1 to the irradiated minipig parotid gland restores fluid secretion
- Z Wang
- , L Zourelias
- & M J Passineau
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Original Article |
Gene electrotransfer of plasmid AMEP, an integrin-targeted therapy, has antitumor and antiangiogenic action in murine B16 melanoma
- M Bosnjak
- , T Dolinsek
- & G Sersa
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Review |
Potential treatments for genetic hearing loss in humans: current conundrums
- R Minoda
- , T Miwa
- & H Takeda
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Original Article |
High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector
- M Quiviger
- , A Arfi
- & C Marie
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Original Article |
Vero/BC-F: an efficient packaging cell line stably expressing F protein to generate single round-infectious human parainfluenza virus type 2 vector
- J Ohtsuka
- , M Fukumura
- & T Nosaka
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Original Article |
Relief of pain induced by varicella-zoster virus in a rat model of post-herpetic neuralgia using a herpes simplex virus vector expressing enkephalin
- J-M G Guedon
- , M Zhang
- & P R Kinchington
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Original Article |
Mammalian orthoreovirus T3D infects U-118 MG cell spheroids independent of junction adhesion molecule-A
- I J C Dautzenberg
- , D J M van den Wollenberg
- & R C Hoeben
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Original Article |
Activation of TLR9 by incorporated pDNA within PEG-coated lipoplex enhances anti-PEG IgM production
- Y Hashimoto
- , Y Uehara
- & H Kiwada
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Original Article |
Safe and efficient local gene delivery into skeletal muscle via a combination of Pluronic L64 and modified electrotransfer
- S Liu
- , L Ma
- & Z Gu
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Original Article |
AAV-mediated expression of human PRELP inhibits complement activation, choroidal neovascularization and deposition of membrane attack complex in mice
- M T Birke
- , E Lipo
- & R Kumar-Singh
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Original Article |
Intranasal administration of plasmid DNA nanoparticles yields successful transfection and expression of a reporter protein in rat brain
- B T Harmon
- , A E Aly
- & B L Waszczak
Primary T cells for mRNA-mediated immunotoxin delivery
Gene therapy research in Asia
Therapeutic approaches for spinal muscular atrophy (SMA)
Developmental regulation of SMN expression: pathophysiological implications and perspectives for therapy development in spinal muscular atrophy
Tackling breast cancer chemoresistance with nano-formulated siRNA
Cell-based gene therapy against HIV