Generation of bidirectional hypoxia/HIF-responsive expression vectors to target gene expression to hypoxic cells.

abstract：:Newcastle disease virus (NDV) is an oncolytic paramyxovirus with a nonsegmented single-stranded RNA genome. In this report, a recombinant oncolytic NDV was passaged in human tumor xenografts and reisolated and characterized after two rounds of bioselection. Several isolates could be recovered that differed from the pa...

journal_title：Gene therapy

authors： Beier R,Hermiston T,Mumberg D

更新日期：2013-01-01 00:00:00

abstract：:Adenovirus vectors have great potential in cancer gene therapy. Targeting of cancer-testis (CT) antigens, which are specifically presented at the surface of tumor cells by human leukocyte antigen (HLA) class I molecules, is an attractive option. In this study, a single-chain T-cell receptor (scTCR) directed against th...

journal_title：Gene therapy

authors： de Vrij J,Uil TG,van den Hengel SK,Cramer SJ,Koppers-Lalic D,Verweij MC,Wiertz EJ,Vellinga J,Willemsen RA,Hoeben RC

更新日期：2008-07-01 00:00:00

abstract：:Chronic infection with hepatitis B virus (HBV) puts individuals at high risk for complicating cirrhosis and liver cancer, but available treatment to counter the virus rarely eliminates infection. Although harnessing RNA interference (RNAi) to silence HBV genes has shown the potential, achieving efficient and durable s...

journal_title：Gene therapy

authors： Ivacik D,Ely A,Ferry N,Arbuthnot P

更新日期：2015-02-01 00:00:00

abstract：:Cell surface targeting of recombinant adeno-associated virus (rAAV) vectors is an attractive strategy to modify AAV's natural tropism. As modification of the capsid surface is likely to affect the mechanism of vector internalization and consequently the vector's intracellular fate, we investigated early steps in cell ...

journal_title：Gene therapy

authors： Uhrig S,Coutelle O,Wiehe T,Perabo L,Hallek M,Büning H

更新日期：2012-02-01 00:00:00

abstract：:This study was designed to evaluate the utility of positron emission tomography (PET) to quantify the magnitude and spatial distribution of transgene expression after different methods of adenoviral vector delivery (with surfactant- and saline-based vehicles) within rat lungs. In all, 17 animals (eight in the surfacta...

journal_title：Gene therapy

authors： Richard JC,Factor P,Welch LC,Schuster DP

更新日期：2003-12-01 00:00:00

abstract：:Self-amplifying RNA (saRNA) is a promising biotherapeutic tool that has been used as a vaccine against both infectious diseases and cancer. saRNA has been shown to induce protein expression for up to 60 days and elicit immune responses with lower dosing than messenger RNA (mRNA). Because saRNA is a large (~9500 nt), n...

journal_title：Gene therapy

authors： Blakney AK,McKay PF,Yus BI,Aldon Y,Shattock RJ

更新日期：2019-09-01 00:00:00

abstract：:Radioiodide concentrating activity in the thyroid, mediated by human Na+/I- symporter (hNIS), provides a mechanism for effective radioiodide treatment for patients who have invasive, recurrent, and metastatic thyroid cancers after total thyroidectomy. In an attempt to develop hNIS gene transfer for radioiodide therapy...

journal_title：Gene therapy

authors： Cho JY,Xing S,Liu X,Buckwalter TL,Hwa L,Sferra TJ,Chiu IM,Jhiang SM

更新日期：2000-05-01 00:00:00

abstract：:Green fluorescent protein (GFP) is a widely used intracellular reporter molecule to assess gene transfer and expression. A potential use for GFP is as a co-expressed marker, to select and enrich gene-modified cells by flow cytometry. Processed peptides derived from GFP and presented by the major histocompatibility com...

journal_title：Gene therapy

authors： Stripecke R,Carmen Villacres M,Skelton D,Satake N,Halene S,Kohn D

更新日期：1999-07-01 00:00:00

abstract：:Inadequate antiprotease activity in the lungs due to alpha-1-antitrypsin (A1AT) deficiency is a factor of early-onset emphysema. We propose a new approach to gene therapy that involves the intratracheal delivery of macrophages expressing human A1AT (hA1AT). Recombinant adeno-associated virus (rAAV) plasmids encoding t...

journal_title：Gene therapy

authors： Zhang D,Wu M,Nelson DE,Pasula R,Martin WJ 2nd

更新日期：2003-12-01 00:00:00

abstract：:Abnormal excitation-contraction coupling is a key pathophysiologic component of heart failure (HF), and at a molecular level reduced expression of the sarcoplasmic reticulum (SR) Ca(2+) ATPase (SERCA2a) is a major contributor. Previous studies in small animals have suggested that restoration of SERCA function is benef...

journal_title：Gene therapy

authors： Byrne MJ,Power JM,Preovolos A,Mariani JA,Hajjar RJ,Kaye DM

更新日期：2008-12-01 00:00:00

abstract：:Introduction of the herpes simplex virus type 1 thymidine kinase gene into tumor cells, followed by the administration of the antiherpes nucleoside analogue ganciclovir has been demonstrated to be effective in eliminating solid tumors in animals. The success of this combination treatment largely depends on the bystand...

journal_title：Gene therapy

authors： Degrève B,De Clercq E,Balzarini J

更新日期：1999-02-01 00:00:00

abstract：:In this study, we describe a simple system in which human keratinocytes can be redirected to an alternative differentiation pathway. We transiently transfected freshly isolated human skin keratinocytes with the single transcription factor OCT4. Within 2 days these cells displayed expression of endogenous embryonic gen...

journal_title：Gene therapy

authors： Racila D,Winter M,Said M,Tomanek-Chalkley A,Wiechert S,Eckert RL,Bickenbach JR

更新日期：2011-03-01 00:00:00

abstract：:Insulin gene therapy in clinical medicine is currently hampered by the inability to regulate insulin secretion in a physiological manner, the inefficiency with which the gene is delivered, and the short duration of gene expression. To address these issues, we injected the liver of streptozotocin-induced diabetic rats ...

journal_title：Gene therapy

authors： Kim YD,Park KG,Morishita R,Kaneda Y,Kim SY,Song DK,Kim HS,Nam CW,Lee HC,Lee KU,Park JY,Kim BW,Kim JG,Lee IK

更新日期：2006-02-01 00:00:00

abstract：:To test whether fast-acting, self-complimentary (sc), adeno-associated virus-mediated RPE65 expression prevents cone degeneration and/or restores cone function, we studied two mouse lines: the Rpe65-deficient rd12 mouse and the Rpe65-deficient, rhodopsin null ('that is, cone function-only') Rpe65(-/-)::Rho(-/-) mouse....

journal_title：Gene therapy

authors： Pang J,Boye SE,Lei B,Boye SL,Everhart D,Ryals R,Umino Y,Rohrer B,Alexander J,Li J,Dai X,Li Q,Chang B,Barlow R,Hauswirth WW

更新日期：2010-07-01 00:00:00

abstract：:Previous studies have demonstrated that antigen-specific tolerance could be induced by lipopolysaccharide (LPS)-stimulated B cells retrovirally transduced with an immunoglobulin-antigen (or epitope-containing peptide) fusion construct. To investigate the mechanism of this gene therapy system, we now adapted this appro...

journal_title：Gene therapy

authors： Song L,Wang J,Wang R,Yu M,Sun Y,Han G,Li Y,Qian J,Scott DW,Kang Y,Soukhareva N,Shen B

更新日期：2004-10-01 00:00:00

abstract：:Most neoplasms do not induce antitumor immune responses that can control tumor growth. Tumor associated antigens (TAAs) are insufficiently immunogenic. A vaccine that augments the immunogenic properties of TAAs could be of importance in the treatment of cancer patients. In an animal model, we prepared a vaccine by tra...

journal_title：Gene therapy

authors： de Zoeten EF,Markovic D,Cohen EP

更新日期：2002-09-01 00:00:00

abstract：:The development of lentiviral vectors that use Rev-independent mechanisms of nuclear export for their genomic RNA could facilitate the construction of novel anti-HIV vectors. We have improved the titers of Rev-independent lentiviral vectors having the SRV-1 CTE by mutating the major splice donor and acceptor sites pre...

journal_title：Gene therapy

authors： Mautino MR,Keiser N,Morgan RA

更新日期：2000-08-01 00:00:00

abstract：:Recently, there has been an increasing level of interest in electroporation for gene delivery due to the site-specific nature of the delivery, as well as the high efficiency of the method. Electroporation involves the application of a pulsed electric field to cells to enhance cell permeability, resulting in the transi...

journal_title：Gene therapy

authors： Liu F,Huang L

更新日期：2002-08-01 00:00:00

abstract：:The failure of pharmacological approaches to cure infection with the human immunodeficiency virus (HIV) has renewed the interest in gene-based therapies. Among the various strategies that are currently explored, the blockade of HIV entry into susceptible T cells and macrophages promises to be the most powerful interve...

journal_title：Gene therapy

authors： Schambach A,Schiedlmeier B,Kühlcke K,Verstegen M,Margison GP,Li Z,Kamino K,Bohne J,Alexandrov A,Hermann FG,von Laer D,Baum C

更新日期：2006-07-01 00:00:00

abstract：:Choroidal neovascularization (CNV) leads to loss of vision in age-related macular degeneration (AMD), the leading cause of blindness in adult population over 50 years old. In this study, we developed intravenously administered, nanoparticulate, targeted nonviral retinal gene delivery systems for the management of CNV....

journal_title：Gene therapy

authors： Singh SR,Grossniklaus HE,Kang SJ,Edelhauser HF,Ambati BK,Kompella UB

更新日期：2009-05-01 00:00:00

abstract：:Isolated limb perfusion (ILP) is a limb salvage surgical modality used to deliver chemotherapy and biologic agents to locally advanced and recurrent extremity soft tissue sarcoma (STS), and may be readily tailored for delivery of gene therapy. We set out to test the feasibility of delivering AdFLAGp53 (replication inc...

journal_title：Gene therapy

authors： Hannay J,Davis JJ,Yu D,Liu J,Fang B,Pollock RE,Lev D

更新日期：2007-04-01 00:00:00

abstract：:Mucopolysaccharidosis type I (MPSI) is an autosomic recessive, lysosomal storage disorder due to the deficit of the enzyme α-L-iduronidase (IDUA). The disease accounts for a general impairment of tissue and organ functions, mainly including heart disease, corneal clouding, organomegaly, skeletal malformations and join...

journal_title：Gene therapy

authors： Piller Puicher E,Tomanin R,Salvalaio M,Friso A,Hortelano G,Marin O,Scarpa M

更新日期：2012-04-01 00:00:00

abstract：:We recently showed that the human telomerase reverse transcriptase (hTERT) promoter induces tumor-specific Bax gene expression and selectively kills various human cancer cells both in vitro and in xenograft tumors. However, it remains unclear whether the hTERT promoter can be used to induce transgene expression in syn...

journal_title：Gene therapy

authors： Gu J,Andreeff M,Roth JA,Fang B

更新日期：2002-01-01 00:00:00

abstract：:Multiple antitumor modalities may be necessary to overcome lung tumor-mediated immunosuppression and effectively treat non-small cell lung cancer (NSCLC). To evaluate a multimodality gene therapy approach for control of local tumor growth, a weakly immunogenic murine alveolar cell carcinoma, L1C2, was transduced with ...

journal_title：Gene therapy

authors： Sharma S,Miller PW,Stolina M,Zhu L,Huang M,Paul RW,Dubinett SM

更新日期：1997-12-01 00:00:00

abstract：:Partial resistance of primary mouse hepatocytes to lentiviral (LV) vector transduction poses a challenge for ex vivo gene therapy protocols in models of monogenetic liver disease. We thus sought to optimize ex vivo LV gene transfer while preserving the hepatocyte integrity for subsequent transplantation into recipient...

journal_title：Gene therapy

authors： Rothe M,Rittelmeyer I,Iken M,Rüdrich U,Schambach A,Glage S,Manns MP,Baum C,Bock M,Ott M,Modlich U

更新日期：2012-04-01 00:00:00

abstract：:Aptamers are oligonucleotides evolved in vitro or in nature to bind target ligands with high affinity and specificity. They are emerging as powerful tools in the fields of therapeutics, drug development, target validation and diagnostics. Aptamers are attractive alternatives to antibody- and small-molecule-based thera...

journal_title：Gene therapy

authors： Que-Gewirth NS,Sullenger BA

更新日期：2007-02-01 00:00:00

abstract：:Dendritic cell (DC)-based vaccines have been applied clinically in the setting of advanced-stage cancer. To date, the clinical efficacy of these vaccines has been limited, possibly owing to the impairment of transferred DC function in cancer-bearing patients. In this study, we examined the therapeutic efficacy of inte...

journal_title：Gene therapy

authors： Tatsumi T,Takehara T,Yamaguchi S,Sasakawa A,Miyagi T,Jinushi M,Sakamori R,Kohga K,Uemura A,Ohkawa K,Storkus WJ,Hayashi N

更新日期：2007-06-01 00:00:00

abstract：:Current generation adenovirus (Ad) vectors are deleted for the E1 region of genes and require propagation in E1 expressing 293 cells. Expression of genes delivered by Ad vectors into immunocompetent hosts is generally transient since the current vectors are not completely replication defective. Viral proteins expresse...

journal_title：Gene therapy

authors： Amalfitano A,Chamberlain JS

更新日期：1997-03-01 00:00:00

abstract：:The proteasome is a multisubunit cytosolic protein complex responsible for degrading cytosolic proteins. Several studies have implicated its involvement in the processing of viral particles used for gene delivery, thereby limiting the efficiency of gene transfer. Peptide-based nonviral gene delivery systems are suffic...

journal_title：Gene therapy

authors： Kim J,Chen CP,Rice KG

更新日期：2005-11-01 00:00:00

abstract：:Cancer cachexia, characterized by anorexia, weight loss and progressive tissue wasting, has been postulated to be mediated by various cytokines. However, the precise mechanism of cachexia induction is not fully explained. We have developed synthetic double-stranded oligodeoxynucleotides (ODN) as 'decoy' cis-elements t...

journal_title：Gene therapy

authors： Kawamura I,Morishita R,Tomita N,Lacey E,Aketa M,Tsujimoto S,Manda T,Tomoi M,Kida I,Higaki J,Kaneda Y,Shimomura K,Ogihara T

更新日期：1999-01-01 00:00:00