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Original Article
| Open AccessTropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina
- D G Hickey
- , T L Edwards
- & R E MacLaren
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Original Article
| Open AccessStrategy to detect pre-existing immunity to AAV gene therapy
- L Falese
- , K Sandza
- & S Fong
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Original Article |
Effects of herpes simplex virus vectors encoding poreless TRPV1 or protein phosphatase 1α in a rat cystitis model induced by hydrogen peroxide
- S Takai
- , T Majima
- & N Yoshimura
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Original Article |
Primary T cells for mRNA-mediated immunotoxin delivery
- R Eggers
- , A Philippi
- & M J Schmitt
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Original Article
| Open AccessEfficacy of combining ING4 and TRAIL genes in cancer-targeting gene virotherapy strategy: first evidence in preclinical hepatocellular carcinoma
- A Galal El-Shemi
- , A Mohammed Ashshi
- & C-O Yun
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Short Communication
| Open AccessTime-series oligonucleotide count to assign antiviral siRNAs with long utility fit in the big data era
- K Wada
- , Y Wada
- & T Ikemura
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Short Communication |
Long-term correction of hemophilia A mice following lentiviral mediated delivery of an optimized canine factor VIII gene
- J M Staber
- , M J Pollpeter
- & P B McCray
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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 |
Stent-based delivery of adeno-associated viral vectors with sustained vascular transduction and iNOS-mediated inhibition of in-stent restenosis
- I Fishbein
- , D T Guerrero
- & R J Levy
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Original Article |
Oligonucleotide-targeting periostin ameliorates pulmonary fibrosis
- A Tomaru
- , T Kobayashi
- & E C Gabazza
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Original Article |
Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler–Najjar Syndrome Type I
- L Bočkor
- , G Bortolussi
- & A F Muro
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Review |
Human genomics projects and precision medicine
- F Carrasco-Ramiro
- , R Peiró-Pastor
- & B Aguado
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Original Article
| Open AccessAAV5-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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Review |
Management of neuromuscular diseases and spinal muscular atrophy in Latin America
- S Monges
- & A L Rosa
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Original Article |
Delivery of human erythropoietin gene with an adeno-associated virus vector through parotid glands to treat renal anaemia in a swine model
- C Ma
- , Z Fan
- & Z Shan
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Short Communication
| Open AccessAAV9-based gene therapy partially ameliorates the clinical phenotype of a mouse model of Leigh syndrome
- I Di Meo
- , S Marchet
- & C Viscomi
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Review
| Open AccessThe importance of international collaboration for rare diseases research: a European perspective
- D Julkowska
- , C P Austin
- & S van Weely
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Original Article |
Nasal application of HSV encoding human preproenkephalin blocks craniofacial pain in a rat model of traumatic brain injury
- A C Meidahl
- , M Klukinov
- & D C Yeomans
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Review |
The state of gene therapy research in Africa, its significance and implications for the future
- P Arbuthnot
- , M B Maepa
- & M S Pepper
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Short Communication |
Gene therapy for spinomuscular atrophy: a biomedical advance, a missed opportunity for more equitable drug pricing
- T Friedmann
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Original Article |
SOST silencing promotes proliferation and invasion and reduces apoptosis of retinoblastoma cells by activating Wnt/β-catenin signaling pathway
- T Wu
- , L-N Wang
- & F-Y Sun
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Review |
Current application of CRISPR/Cas9 gene-editing technique to eradication of HIV/AIDS
- Z Huang
- , A Tomitaka
- & M Nair
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Original Article |
Silencing of HIV-1 by AgoshRNA molecules
- E Herrera-Carrillo
- , A Harwig
- & B Berkhout
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Short Communication |
Antigen-presenting cell-targeted lentiviral vectors do not support the development of productive T-cell effector responses: implications for in vivo targeted vaccine delivery
- C Goyvaerts
- , Y De Vlaeminck
- & K Breckpot
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Original Article |
Potential mechanisms of microRNA-141-3p to alleviate chronic inflammatory pain by downregulation of downstream target gene HMGB1: in vitro and in vivo studies
- W-S Shen
- , X-Q Xu
- & Y-H Yu
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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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Short Communication |
Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders
- K Bey
- , C Ciron
- & M-A Colle
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Original Article |
MnSOD mediated by HSV vectors in the periaqueductal gray suppresses morphine withdrawal in rats
- T Iida
- , H Yi
- & S Hao
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Original Article |
Optimization of adeno-associated virus vector-mediated gene transfer to the respiratory tract
- F Kurosaki
- , R Uchibori
- & A Kume
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Original Article |
IL-4 mediated by HSV vector suppresses morphine withdrawal response and decreases TNFα, NR2B, and pC/EBPβ in the periaqueductal gray in rats
- H Yi
- , T Iida
- & S Hao
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Short Communication
| Open AccessMR-guided parenchymal delivery of adeno-associated viral vector serotype 5 in non-human primate brain
- L Samaranch
- , B Blits
- & K S Bankiewicz
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Original Article |
Modulation of digital flexor tendon healing by vascular endothelial growth factor gene transfection in a chicken model
- W F Mao
- , Y F Wu
- & J B Tang
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Original Article |
An AAVrh10-CAG-CYP21-HA vector allows persistent correction of 21-hydroxylase deficiency in a Cyp21−/− mouse model
- M Perdomini
- , C Dos Santos
- & P Bougnères
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Short Communication |
Neutralizing antibodies against adeno-associated viruses in Sjögren’s patients: implications for gene therapy
- A Corden
- , B Handelman
- & J A Chiorini
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Original Article
| Open AccessExtraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression
- M L Katz
- , G C Johnson
- & M S Whitney
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Review |
Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems
- S Guan
- & J Rosenecker
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Comment |
Delivering efficient liver-directed AAV-mediated gene therapy
- J Baruteau
- , S N Waddington
- & P Gissen
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Original Article
| Open AccessBalanced secretion of anti-CEA × anti-CD3 diabody chains using the 2A self-cleaving peptide maximizes diabody assembly and tumor-specific cytotoxicity
- K Mølgaard
- , M Compte
- & L Alvarez-Vallina
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News and Commentary |
Gene therapy approaches for prevention of retinal degeneration in Usher syndrome
- D S Williams
- , A Chadha
- & D Gibbs
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Original Article |
Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich ataxia
- D L Ouellet
- , K Cherif
- & J P Tremblay
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Original Article |
Evidence for the in vivo safety of insulated foamy viral vectors
- D L Browning
- , E M Everson
- & G D Trobridge
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