Targeted chemotherapy by intratumour injection of encapsulated cells engineered to produce CYP2B1, an ifosfamide activating cytochrome P450.

abstract：:Chimeric antigen receptors (CARs, immunoreceptors) are frequently used to redirect T cells with pre-defined specificity, in particular towards tumour cells for use in adoptive immunotherapy of malignant diseases. Specific targeting is mediated by an extracellularly located antibody-derived binding domain, which is joi...

journal_title：Gene therapy

authors： Hombach A,Hombach AA,Abken H

更新日期：2010-10-01 00:00:00

abstract：:Gene modification of malignant cells to express immune stimulators (cytokines and immune costimulators) has provided the basis for a novel form of immunotherapy. Using a MPSV-based retroviral vector with hygromycin resistance gene as a selectable marker, we have studied retrovirus-mediated gene transfer of an immune c...

journal_title：Gene therapy

authors： Hirst WJ,Buggins A,Darling D,Gäken J,Farzaneh F,Mufti GJ

更新日期：1997-07-01 00:00:00

abstract：:The possibility of achieving multiple systemic expression of human interferon-beta in mice upon repeated intravenous administration of cationic liposome-DNA complex (lipoplex) was investigated. Lipoplexes containing the pentammonio lipid pcTG90 were first optimized by selecting the most efficient ratio of pcTG90 to ph...

journal_title：Gene therapy

authors： Meyer O,Schughart K,Pavirani A,Kolbe HV

更新日期：2000-09-01 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：:To investigate to what extent myeloablation, graft size, and ex vivo manipulation influence the engraftment and long-term survival of transduced murine hematopoietic cells, groups of C57BL/6J (CD45.2) mice receiving total body irradiation (TBI) (1-9 Gy) or no irradiation were transplanted with either transduced bone m...

journal_title：Gene therapy

authors： Puig T,Kádár E,Limón A,Cancelas JA,Eixarch H,Luquín L,García M,Barquinero J

更新日期：2002-11-01 00:00:00

abstract：:To confer adenovirus vectors (AdV), the feature of integration into the host cell genome hybrid vectors were characterized in vitro, which express vectors derived from the prototypic foamy virus (FV) in the backbone of a high-capacity AdV. FVs constitute a subfamily of retroviruses with a distinct replication pathway ...

journal_title：Gene therapy

authors： Picard-Maureau M,Kreppel F,Lindemann D,Juretzek T,Herchenröder O,Rethwilm A,Kochanek S,Heinkelein M

更新日期：2004-04-01 00:00:00

abstract：:Exogenous osteoprotegerin (OPG) gene modification appears a therapeutic strategy for osteolytic aseptic loosening. The feasibility and efficacy of a cell-based OPG gene delivery approach were investigated using a murine model of knee prosthesis failure. A titanium pin was implanted into mouse proximal tibia to mimic a...

journal_title：Gene therapy

authors： Zhang L,Jia TH,Chong AC,Bai L,Yu H,Gong W,Wooley PH,Yang SY

更新日期：2010-10-01 00:00:00

abstract：:Chimeric antigen receptor (CAR) T-cell therapies have demonstrated durable and potentially curative therapeutic efficacy against B-cell leukemia in clinical trials. A CAR strategy can target any tumor surface antigens as long as an antigen-binding receptor can be generated. New CARs that target solid tumors and have t...

journal_title：Gene therapy

authors： Wu MR,Zhang T,DeMars LR,Sentman CL

更新日期：2015-08-01 00:00:00

abstract：:For certain gene therapy applications, the simultaneous delivery of multiple genes would allow for novel therapies. In the case of adeno-associated virus (AAV) vectors, the limited packaging capacity greatly restricts current methods of carrying multiple transgene cassettes. To address this issue, a recombinant AAV (r...

journal_title：Gene therapy

authors： Foti SB,Samulski RJ,McCown TJ

更新日期：2009-11-01 00:00:00

abstract：:Until recently, adeno-associated virus 9 (AAV9) was considered the AAV serotype most effective in crossing the blood-brain barrier (BBB) and transducing cells of the central nervous system (CNS), following systemic injection. However, a newly engineered capsid, AAV-PHP.B, is reported to cross the BBB at even higher ef...

journal_title：Gene therapy

authors： Rincon MY,de Vin F,Duqué SI,Fripont S,Castaldo SA,Bouhuijzen-Wenger J,Holt MG

更新日期：2018-04-01 00:00:00

abstract：:To achieve high transgene expression in the liver, we have compared the reporter gene expression among various murine retroviral long terminal repeats (LTRs) or leader sequences in vitro. Transient reporter gene expression assays revealed the highest gene expression by the polycythemic strain of spleen focus-forming v...

journal_title：Gene therapy

authors： Ohnishi N,Itoh K,Itoh Y,Baum C,Higashitsuji H,Yamaguchi K,Tsuji T,Okanoue T,Fujita J

更新日期：2002-02-01 00:00:00

abstract：:This study examines the clinical relevance of tissue engineering integrating gene therapy and polymer science to bone regeneration. Bilateral maxillary defects (3 x 1.2 cm(2)) in 20 miniature swine were bridged with a bioresorbable internal splint. Constructs were created using ex vivo adenovirus bone morphogenetic pr...

journal_title：Gene therapy

authors： Chang SC,Chuang HL,Chen YR,Chen JK,Chung HY,Lu YL,Lin HY,Tai CL,Lou J

更新日期：2003-11-01 00:00:00

abstract：:Human adipose tissue mesenchymal stromal cells (AMSCs) share common traits, including similar differentiation potential and cell surface markers, with their bone marrow counterparts. Owing to their general availability, higher abundance and ease of isolation AMSCs may be convenient autologous delivery vehicles for loc...

journal_title：Gene therapy

authors： Vilalta M,Dégano IR,Bagó J,Aguilar E,Gambhir SS,Rubio N,Blanco J

更新日期：2009-04-01 00:00:00

abstract：:Highly active antiretroviral therapy (HAART) treatment for HIV has changed the course of AIDS in societies in which the drugs are readily available. Despite the great success of HAART, drug resistance and toxicity issues still remain a concern for some individuals. Thus, a number of investigators have been exploring o...

journal_title：Gene therapy

authors： Zhou J,Rossi JJ

更新日期：2011-12-01 00:00:00

abstract：:Hypoxia initiates an adaptive physiological response in all organisms and plays a role in the pathogenesis of several human diseases. The hypoxia/HIF-inducible factor-1 (HIF-1) transcription factor mediates transcriptional responses to hypoxia by binding to a cis-acting hypoxia-responsive element (HRE) present within ...

journal_title：Gene therapy

authors： Post DE,Van Meir EG

更新日期：2001-12-01 00:00:00

abstract：:Rescue of dystrophic skeletal muscle in mdx and utrophin/dystrophin-deficient (dko) mouse models by reintroduction of dystrophin has validated gene therapy as a potential therapeutic approach for Duchenne muscular dystrophy. However, the size of the dystrophin gene exceeds the capacity of adeno-associated viral (AAV) ...

journal_title：Gene therapy

authors： Gardner KL,Kearney JA,Edwards JD,Rafael-Fortney JA

更新日期：2006-05-01 00:00:00

abstract：:Platelet-derived growth factor-BB (PDGF-BB) stimulates repair of healing-impaired chronic wounds such as diabetic ulcers and periodontal lesions. However, limitations in predictability of tissue regeneration occur due, in part, to transient growth factor bioavailability in vivo. Here, we report that gene delivery of P...

journal_title：Gene therapy

authors： Chang PC,Seol YJ,Cirelli JA,Pellegrini G,Jin Q,Franco LM,Goldstein SA,Chandler LA,Sosnowski B,Giannobile WV

更新日期：2010-01-01 00:00:00

abstract：:Recently developed helper virus-free methods of herpes simplex virus (HSV) amplicon vector packaging provide stocks that are virtually devoid of the cytotoxic component normally associated with traditional helper virus-based packaging methods. These approaches involve cotransfection of amplicon plasmid DNA with either...

journal_title：Gene therapy

authors： Bowers WJ,Howard DF,Brooks AI,Halterman MW,Federoff HJ

更新日期：2001-01-01 00:00:00

abstract：:We have used a particular folate receptor, which is overexpressed in tumor cells, for targeted DNA delivery into these cell types. This folate receptor internalizes folate through caveolae by a process named potocytosis, which is distinct from endocytosis, through clathrin-coated pits. When folate conjugated to poly-L...

journal_title：Gene therapy

authors： Gottschalk S,Cristiano RJ,Smith LC,Woo SL

更新日期：1994-05-01 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：:A replication-incompetent adenoviral vector encoding the heavy chain C-fragment (H(C)50) of botulinum neurotoxin type C (BoNT/C) was evaluated as a mucosal vaccine against botulism in a mouse model. Single intranasal inoculation of the adenoviral vector elicited a high level of H(C)50-specific IgG, IgG1 and IgG2a in s...

journal_title：Gene therapy

authors： Xu Q,Pichichero ME,Simpson LL,Elias M,Smith LA,Zeng M

更新日期：2009-03-01 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：:To identify the intracellular barriers to efficient gene transfer, we studied the intracellular trafficking of biotinylated plasmid DNA complexed with either fluorescein-conjugated lactosylated or mannosylated polylysine by confocal microscopy. Both are known to be taken up by cystic fibrosis airway epithelial cells (...

journal_title：Gene therapy

authors： Grosse S,Tremeau-Bravard A,Aron Y,Briand P,Fajac I

更新日期：2002-08-01 00:00:00

abstract：:Defects of the mitochondrial genome cause a wide variety of clinical disorders. Except for rare cases where surgery or transplant is indicated, there is no effective treatment for patients. Genetic-based therapies are consequently being considered. On account of the difficulties associated with mitochondrial (mt) tran...

journal_title：Gene therapy

authors： Kyriakouli DS,Boesch P,Taylor RW,Lightowlers RN

更新日期：2008-07-01 00:00:00

abstract：:Several groups are assessing the use of cationic lipids for respiratory gene therapy. To date no human data are available regarding the safety of intra-pulmonary cationic lipid delivery. In preparation for a trial of pulmonary delivery of the CFTR gene, we have assessed the safety of nebulised lipid GL-67/DOPE/DMPE-PE...

journal_title：Gene therapy

authors： Chadwick SL,Kingston HD,Stern M,Cook RM,O'Connor BJ,Lukasson M,Balfour RP,Rosenberg M,Cheng SH,Smith AE,Meeker DP,Geddes DM,Alton EW

更新日期：1997-09-01 00:00:00

abstract：:Galactose(alpha1,3)galactose on the surface of cells of non-primate organs is the major xenoantigen responsible for hyperacute rejection in xenotransplantation. The antigen is synthesised by (alpha1, 3)galactosyl transferase. Humans lack this enzyme and their serum contains high levels of pre-existing natural antibody...

journal_title：Gene therapy

authors： Jäger U,Takeuchi Y,Porter C

更新日期：1999-06-01 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：:We report on long-term delivery of an interferon-gamma (IFN gamma) inhibitory protein by intramuscular (i.m.) gene therapy. IFN gamma is a cytokine that plays an important role in many inflammatory disorders, including autoimmune insulin-dependent diabetes mellitus (IDDM) in NOD mice and (in various strains) multiple ...

journal_title：Gene therapy

authors： Prud'homme GJ,Chang Y

更新日期：1999-05-01 00:00:00

abstract：:We demonstrated previously that the additive-type recombinant Sendai virus (rSeV) is highly efficient for use in pulmonary gene transfer; however, rSeV exhibits inflammatory responses. To overcome this problem, we tested newly developed non-transmissible constructs, namely, temperature-sensitive F-deleted vector, rSeV...

journal_title：Gene therapy

authors： Tanaka S,Yonemitsu Y,Yoshida K,Okano S,Kondo H,Inoue M,Hasegawa M,Masumoto K,Suita S,Taguchi T,Sueishi K

更新日期：2007-07-01 00:00:00

abstract：:In this study, self-inactivating (SIN) retroviral vectors based on feline foamy virus (FFV) were constructed and analysed. The FFV SIN vectors were devoid of the core FFV long terminal repeat promoter plus upstream sequences but contained all structural and regulatory genes. This design allowed sensitive detection of ...

journal_title：Gene therapy

authors： Bastone P,Löchelt M

更新日期：2004-03-01 00:00:00