Featured
-
-
Article |
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
- Colin L. Sweeney
- , Mara Pavel-Dinu
- & Suk See De Ravin
-
Article
| Open Access
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells
- Anna K. Dreismann
- , Michelle E. McClements
- & Robert E. MacLaren
-
Article |
Lentiviral gene therapy vectors encoding VIP suppressed diabetes-related inflammation and augmented pancreatic beta-cell proliferation
- Fulya Erendor
- , Elif Ozgecan Sahin
- & Salih Sanlioglu
-
Brief Communication
| Open Access
Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice
- Andrea Schejtman
- , Walmir Cutrim Aragão-Filho
- & Giorgia Santilli
-
Brief Communication |
Lentiviral gene therapy and vitamin B3 treatment enable granulocytic differentiation of G6PC3-deficient induced pluripotent stem cells
- Dirk Hoffmann
- , Johannes Kuehle
- & Axel Schambach
-
Article
| Open Access
Immunosuppression overcomes insulin- and vector-specific immune responses that limit efficacy of AAV2/8-mediated insulin gene therapy in NOD mice
- Asha Recino
- , Shu Uin Gan
- & Maja Wallberg
-
-
Brief Communication |
HIV-based lentivirus-mediated vasoactive intestinal peptide gene delivery protects against DIO animal model of Type 2 diabetes
- Hale M. Tasyurek
- , Yunus E. Eksi
- & Salih Sanlioglu
-
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
-
Review |
Current application of CRISPR/Cas9 gene-editing technique to eradication of HIV/AIDS
- Z Huang
- , A Tomitaka
- & M Nair
-
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
-
Short Communication |
Neutralizing antibodies against adeno-associated viruses in Sjögren’s patients: implications for gene therapy
- A Corden
- , B Handelman
- & J A Chiorini
-
Short Communication |
Co-delivery of indoleamine 2,3-dioxygenase prevents loss of expression of an antigenic transgene in dystrophic mouse muscles
- D Sharma
- , R Al-Khalidi
- & D C Gorecki
-
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
-
Original Article |
A novel intranuclear RNA vector system for long-term stem cell modification
- Y Ikeda
- , A Makino
- & K Tomonaga
-
Original Article |
BTK gene targeting by homologous recombination using a helper-dependent adenovirus/adeno-associated virus hybrid vector
- H Yamamoto
- , M Ishimura
- & T Hara
-
Original Article |
Gene therapy with neurogenin3, betacellulin and SOCS1 reverses diabetes in NOD mice
- R Li
- , E Buras
- & L Chan
-
-
Original Article |
A novel gene therapy strategy using secreted multifunctional anti-HIV proteins to confer protection to gene-modified and unmodified target cells
- A Falkenhagen
- , M Ameli
- & S Joshi
-
Mini Review |
Current progress on gene therapy for primary immunodeficiencies
- L Zhang
- , A J Thrasher
- & H B Gaspar
-
Short Communication |
The immunogenicity of virus-derived 2A sequences in immunocompetent individuals
- C Arber
- , H Abhyankar
- & B Savoldo
-
Original Article
| Open Access
Inhibition of neointimal hyperplasia in a rabbit vein graft model following non-viral transfection with human iNOS cDNA
- Q-H Meng
- , S Irvine
- & S L Hart
-
Original Article |
Expression of HSV-1 receptors in EBV-associated lymphoproliferative disease determines susceptibility to oncolytic HSV
- P-Y Wang
- , M A Currier
- & T P Cripe
-
Original Article |
Transcriptional targeting of DCs with lentiviral vectors induces antigen-specific tolerance in a mouse model of multiple sclerosis
- B de Andrade Pereira
- , C Fraefel
- & C Dresch
-
Original Article |
Regulation of immunological balance by sustained interferon-γ gene transfer for acute phase of atopic dermatitis in mice
- K Watcharanurak
- , M Nishikawa
- & Y Takakura
-
Original Article |
Effector memory and central memory NY-ESO-1-specific re-directed T cells for treatment of multiple myeloma
- P C Schuberth
- , G Jakka
- & U Petrausch
-
Short Communication |
Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency
- J C M van der Loo
- , W P Swaney
- & P Malik
-
Original Article |
Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott–Aldrich syndrome
- M Catucci
- , F Prete
- & A Villa
-
Original Article |
Hydrodynamic delivery of plasmid DNA encoding human FcγR-Ig dimers blocks immune-complex mediated inflammation in mice
- R Shashidharamurthy
- , D Machiah
- & P Selvaraj
-
Original Article |
Non-myeloablative transplantation of bone marrow expressing self-antigen establishes peripheral tolerance and completely prevents autoimmunity in mice
- H Hosseini
- , D Y Oh
- & J Chan
-
Review |
Current progress in the development of RNAi-based therapeutics for HIV-1
- J Zhou
- & J J Rossi
-
Original Article |
Lineage- and stage-restricted lentiviral vectors for the gene therapy of chronic granulomatous disease
- I Barde
- , E Laurenti
- & D Trono
-
Original Article |
Early gestational gene transfer of IL-10 by systemic administration of lentiviral vector can prevent arthritis in a murine model
- J L Roybal
- , M Endo
- & A W Flake
-
Original Article |
Treatment of experimental autoimmune encephalomyelitis in SJL/J mice with a replicative HSV-1 vector expressing interleukin-5
- M Nygårdas
- , C Aspelin
- & V Hukkanen
-
Original Article |
Treatment of canine leukocyte adhesion deficiency by foamy virus vectors expressing CD18 from a PGK promoter
- T R Bauer Jr
- , E M Olson
- & D W Russell
-
Original Article |
Anti-inflammatory effect of MAPK phosphatase-1 local gene transfer in inflammatory bone loss
- H Yu
- , Q Li
- & K L Kirkwood
-
Original Article |
MVA-nef induces HIV-1-specific polyfunctional and proliferative T-cell responses revealed by the combination of short- and long-term immune assays
- S Kutscher
- , S Allgayer
- & A Cosma
-
Original Article |
Intra-articular lentivirus-mediated delivery of galectin-3 shRNA and galectin-1 gene ameliorates collagen-induced arthritis
- C-R Wang
- , A-L Shiau
- & C-L Wu
-
Review |
Progress and prospects: graft-versus-host disease
- S Mastaglio
- , M T L Stanghellini
- & C Bonini
-
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
-
Enabling Technologies |
Mass spectrometry measurement of a therapeutic peptide for use in multiple sclerosis
- J M Dadgari
- , R E Moore
- & M McMillan
-
Original Article |
Cytokine gene-modulated dendritic cells protect against allergic airway inflammation by inducing IL-10+IFN-γ+CD4+ T cells
- C-Y Hsu
- , S-J Leu
- & Y-L Lee
-
Short Communication |
Anti-inflammatory effect by lentiviral-mediated overexpression of IL-10 or IL-1 receptor antagonist in rat glial cells and macrophages
- M E van Strien
- , D Mercier
- & A-M van Dam
-
Short Communication |
Lentiviral vectors incorporating a human elongation factor 1α promoter for the treatment of canine leukocyte adhesion deficiency
- E J R Nelson
- , L M Tuschong
- & D D Hickstein
HIV infection alters the human epigenetic landscape
Cell-based gene therapy against HIV