Significant behavioral recovery in Parkinson's disease model by direct intracerebral gene transfer using continuous injection of a plasmid DNA-liposome complex.

abstract：:Interleukin (IL)-12 has been reported to induce cellular immune responses for protection against tumor formation. Here we investigate the utility of adenoviral delivery of IL-12 as an adjuvant for a human papillomavirus E7 subunit vaccine in a mouse tumor challenge model. Direct intratumoral injection of AdIL-12 resul...

journal_title：Human gene therapy

authors： Ahn WS,Bae SM,Kim TY,Kim TG,Lee JM,Namkoong SE,Kim CK,Sin JI

更新日期：2003-10-10 00:00:00

abstract：:Recombinant adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great potential for human gene therapy. One major challenge for translation of promising research to clinical development is the manufacture of sufficient quantities of AAV vectors that meet stringent standards for p...

journal_title：Human gene therapy

authors： Wright JF

更新日期：2009-07-01 00:00:00

abstract：:The epithelial-mesenchymal transition (EMT) has been recognized to occur during embryonic development, fibrosis, and tumor metastasis. Nuclear factor (NF)-κB plays a central role in mediating the inflammation and wound-healing responses during liver fibrogenesis. However, the involvement of NF-κB during EMT in liver c...

journal_title：Human gene therapy

authors： Kim KH,Lee WR,Kang YN,Chang YC,Park KK

更新日期：2014-08-01 00:00:00

abstract：:Liver ischemia-reperfusion (I/R) injury is a multifactorial process that affects graft function after liver transplantation. Inflammatory cytokines, such as tumor necrosis factor (TNF)-α, interleukin (IL)-6, IL-1β, and IL-18, have been shown to play key roles in the pathophysiology of liver I/R injury. Studies have in...

journal_title：Human gene therapy

authors： Zhu P,Duan L,Chen J,Xiong A,Xu Q,Zhang H,Zheng F,Tan Z,Gong F,Fang M

更新日期：2011-07-01 00:00:00

abstract：:Replication-deficient adenovirus vector (Ad) is one of the most efficient gene transfer vehicles for human gene therapy. However, Ad is antigenic, known to evoke prominent inflammatory responses in vivo, and there are concerns that using Ad in patients with immune-mediated disorders (allergy and autoimmune diseases) m...

journal_title：Human gene therapy

authors： Suzuki M,Suzuki S,Yamamoto N,Komatsu S,Inoue S,Hashiba T,Nishikawa M,Ishigatsubo Y

更新日期：2000-04-10 00:00:00

abstract：:One of the major obstacles to pulmonary-directed gene therapy using adenoviral vectors is the induction of inflammation. We investigated whether the adenoviral particles that constitute the initial inoculum can serve as an inflammatory stimulus, independent of their ability to express genes that they contain. Viral pa...

journal_title：Human gene therapy

authors： McCoy RD,Davidson BL,Roessler BJ,Huffnagle GB,Janich SL,Laing TJ,Simon RH

更新日期：1995-12-01 00:00:00

abstract：:Interleukin-12 (IL-12) is a cytokine that exhibits pleiotropic effects on lymphocytes and natural killer cells and has been shown to have promise for the immunotherapy of cancer. The combination of the immune costimulatory molecule B7.1 and IL-12 has been shown to be synergistic for T cell activation. By transfecting ...

journal_title：Human gene therapy

authors： Anderson R,Macdonald I,Corbett T,Hacking G,Lowdell MW,Prentice HG

更新日期：1997-06-10 00:00:00

abstract：:Kallistatin is a serine proteinase inhibitor that has been shown to reduce joint swelling and to inhibit inflammation in a rat model of arthritis. In this study, we investigated the effect and mechanisms of kallistatin on cardiac function after myocardial ischemia-reperfusion (I/R) injury. The human kallistatin gene i...

journal_title：Human gene therapy

authors： Chao J,Yin H,Yao YY,Shen B,Smith RS Jr,Chao L

更新日期：2006-12-01 00:00:00

abstract：:Human bocavirus type-1 (HBoV1) has a high tropism for the apical membrane of human airway epithelia. The packaging of a recombinant adeno-associated virus 2 (rAAV2) genome into HBoV1 capsid produces a chimeric vector (rAAV2/HBoV1) that also efficiently transduces human airway epithelia. As such, this vector is attract...

journal_title：Human gene therapy

authors： Yan Z,Feng Z,Sun X,Zhang Y,Zou W,Wang Z,Jensen-Cody C,Liang B,Park SY,Qiu J,Engelhardt JF

更新日期：2017-08-01 00:00:00

abstract：:Niemann-Pick type C (NP-C) disease is a neurodegenerative disorder characterized neuropathologically by ballooned neurons distended with lipid storage and widespread neuronal loss. Neural stem cells (NSC) derived from NP-C disease models have decreased ability for self-renewal and neuronal differentiation. Investigati...

journal_title：Human gene therapy

authors： Lee H,Kang JE,Lee JK,Bae JS,Jin HK

更新日期：2013-07-01 00:00:00

abstract：:Following in vivo recombinant adeno-associated virus (rAAV)-based gene transfer, adaptive immune responses specific to the vector or the transgene product have emerged as a potential roadblock to successful clinical translation. The occurrence of such responses depends on several parameters, including the route of vec...

journal_title：Human gene therapy

authors： Gernoux G,Guilbaud M,Dubreil L,Larcher T,Babarit C,Ledevin M,Jaulin N,Planel P,Moullier P,Adjali O

更新日期：2015-01-01 00:00:00

abstract：:Replication-deficient viral vectors are currently being used in gene transfer strategies to treat cancer cells. Unfortunately, viruses are limited in their ability to diffuse through tissue. This makes it virtually impossible to infect the majority of tumor cells in vivo and results in inadequate gene transfer. This p...

journal_title：Human gene therapy

authors： Han JS,Qian D,Wicha MS,Clarke MF

更新日期：1998-05-20 00:00:00

abstract：:Dystrophic epidermolysis bullosa (DEB) comprises a family of inherited blistering skin disorders for which no corrective therapy currently exists. In the most severe form, the Hallopeau-Siemens subtype (RDEB-HS), the epidermal adhesion protein collagen VII is absent from the skin as a consequence of null mutations in ...

journal_title：Human gene therapy

authors： Mecklenbeck S,Compton SH,Mejía JE,Cervini R,Hovnanian A,Bruckner-Tuderman L,Barrandon Y

更新日期：2002-09-01 00:00:00

abstract：:The use of synthetic gene delivery systems in human gene transfer is hampered by poor transfection efficiencies, largely because of the inability of DNA to translocate across the nuclear pore complex. A means to overcome this barrier is to bind the DNA to nuclear localization signals (NLSs), which are recognized by sh...

journal_title：Human gene therapy

authors： Mesika A,Kiss V,Brumfeld V,Ghosh G,Reich Z

更新日期：2005-02-01 00:00:00

abstract：:In previous studies we demonstrated that a modified human HSP70b promoter (HSE.70b) directs high levels of gene expression to tumor cells after mild hyperthermia treatment in the range of 41.5-44 degrees C. This transcriptional targeting system exhibits low basal activity at 37 degrees C, is highly induced (950-fold) ...

journal_title：Human gene therapy

authors： Brade AM,Szmitko P,Ngo D,Liu FF,Klamut HJ

更新日期：2003-03-20 00:00:00

abstract：:Leukocyte adhesion deficiency type I (LAD-I) is a primary immunodeficiency caused by mutations in the ITGB2 gene and is characterized by recurrent and life-threatening bacterial infections. These mutations lead to defective or absent expression of β2 integrins on the leukocyte surface, compromising adhesion and extrav...

journal_title：Human gene therapy

authors： Leon-Rico D,Aldea M,Sanchez-Baltasar R,Mesa-Nuñez C,Record J,Burns SO,Santilli G,Thrasher AJ,Bueren JA,Almarza E

更新日期：2016-09-01 00:00:00

abstract：:Atrogin-1 or muscle atrophy F-box (MAFbx) is a major atrophy-related E3 ubiquitin ligase highly expressed in skeletal muscle during muscle atrophy and other disease states such as sepsis, cancer cachexia, and fasting. In this paper, we report experiments inhibiting MAFbx activity in fasting mice and in the skeletal my...

journal_title：Human gene therapy

authors： Cong H,Sun L,Liu C,Tien P

更新日期：2011-03-01 00:00:00

abstract：:The potential of short interfering RNA (siRNA) to be developed for therapeutic use against cancer depends on the availability of an efficient tumor-specific delivery vehicle. We have previously shown that a nanoscale nonviral liposome-based complex that includes an anti-transferrin receptor single-chain antibody fragm...

journal_title：Human gene therapy

authors： Pirollo KF,Zon G,Rait A,Zhou Q,Yu W,Hogrefe R,Chang EH

更新日期：2006-01-01 00:00:00

abstract：:Severe fetal growth restriction (FGR) affects 1:500 pregnancies, is untreatable and causes serious neonatal morbidity and death. Reduced uterine blood flow (UBF) and lack of bioavailable VEGF due to placental insufficiency is a major cause. Transduction of uterine arteries in normal or FGR sheep and guinea pigs using ...

journal_title：Human gene therapy

authors： Rossi C,Lees M,Mehta V,Heikura T,Martin J,Zachary I,Spencer R,Peebles DM,Shaw R,Karhinen M,Yla-Herttuala S,David AL

更新日期：2020-11-01 00:00:00

abstract：:Activation of T cells is necessary for efficient retroviral-mediated gene transfer. In addition, if the population of infused cells is to be limited to transduced cells, a means of positive selection is required. We describe a clinical scale procedure for activation of donor T cells with anti-CD3/CD28 beads followed b...

journal_title：Human gene therapy

authors： Orchard PJ,Blazar BR,Burger S,Levine B,Basso L,Nelson DM,Gordon K,McIvor RS,Wagner JE,Miller JS

更新日期：2002-05-20 00:00:00

abstract：:Ornithine transcarbamylase deficiency (OTCD) is an inborn error of urea synthesis that has been considered as a model for liver-directed gene therapy. Current treatment has failed to avert a high mortality or morbidity from hyperammonemic coma. Restoration of enzyme activity in the liver should suffice to normalize me...

journal_title：Human gene therapy

authors： Raper SE,Yudkoff M,Chirmule N,Gao GP,Nunes F,Haskal ZJ,Furth EE,Propert KJ,Robinson MB,Magosin S,Simoes H,Speicher L,Hughes J,Tazelaar J,Wivel NA,Wilson JM,Batshaw ML

更新日期：2002-01-01 00:00:00

abstract：:The field of adenovirology is undergoing rapid change in response to increasing appreciation of the potential advantages of adenoviruses as the basis for new vaccines and as vectors for gene and cancer therapy. Substantial knowledge and understanding of adenoviruses at a molecular level has made their manipulation for...

journal_title：Human gene therapy

authors： Majhen D,Calderon H,Chandra N,Fajardo CA,Rajan A,Alemany R,Custers J

更新日期：2014-04-01 00:00:00

abstract：:Retrovirus integration into the host cell genome occurs most efficiently in replicating cells. In agreement with this notion, it was observed that the efficiency with which hemopoietic stem cells (HSC) can be transduced is greatly enhanced when the hemopoietic growth factor (HGF) interleukin 3 (IL-3) is added to co-cu...

journal_title：Human gene therapy

authors： Einerhand MP,Bakx TA,Valerio D

更新日期：1991-01-01 00:00:00

abstract：:Three retroviral constructs containing a full-length human alpha-L-iduronidase (IDUA) cDNA were made. The first, pLIdSN, is designed so that expression of the IDUA cDNA is from the 5' viral long terminal repeat (LTR). The second, pLNCId, is designed to express the IDUA cDNA from the cytomegalovirus (CMV) immediate ear...

journal_title：Human gene therapy

authors： Anson DS,Bielicki J,Hopwood JJ

更新日期：1992-08-01 00:00:00

abstract：:Airway infiltration by eosinophils is a major characteristic of chronic asthma. CCL11 (eotaxin-1) is secreted by lung epithelial cells and functions as the major chemokine for eosinophil recruitment. Pseudotyped adeno-associated virus (AAV) 2/9, composed by the AAV2 rep and AAV9 cap genes, can efficiently target lung ...

journal_title：Human gene therapy

authors： Wu CJ,Huang WC,Chen LC,Shen CR,Kuo ML

更新日期：2012-11-01 00:00:00

abstract：:Duchenne muscular dystrophy (DMD) and other inherited myopathies lead to progressive destruction of most skeletal muscles in the body, including those responsible for maintaining respiration. DMD is a fatal disorder caused by defects in the dystrophin gene. Recombinant adenovirus vectors (AdV) are considered a promisi...

journal_title：Human gene therapy

authors： Cho WK,Ebihara S,Nalbantoglu J,Gilbert R,Massie B,Holland P,Karpati G,Petrof BJ

更新日期：2000-03-20 00:00:00

abstract：:The use of viral thymidine kinase (TK) gene coupled with the administration of ganciclovir to render cancer cell death has been studied extensively. Many of these experiments utilized retrovirus to transfer the TK gene under the control of a nonspecific promoter. Because nonspecific expression of the viral TK gene may...

journal_title：Human gene therapy

authors： Su H,Chang JC,Xu SM,Kan YW

更新日期：1996-03-01 00:00:00

abstract：:Subcutaneous vaccination therapy with glioma cells, which are retrovirally transduced to secrete granulocyte-macrophage colony-stimulating factor (GM-CSF), has previously proven effective in C57BL/6 mice harboring intracerebral GL261 gliomas. However, clinical ex vivo gene therapy for human gliomas would be difficult,...

journal_title：Human gene therapy

authors： Herrlinger U,Jacobs A,Quinones A,Woiciechowsky C,Sena-Esteves M,Rainov NG,Fraefel C,Breakefield XO

更新日期：2000-07-01 00:00:00

abstract：:Adenovirus (Ad) vectors used for gene therapy are efficient in entering the infected cell and targeting their genome to the nucleus. To study the mechanism of the interaction between Ad and the nuclear envelope we have established an in vitro assay using rat liver nuclei incubated with serotype 5 Ad vector. Binding of...

journal_title：Human gene therapy

authors： Wisnivesky JP,Leopold PL,Crystal RG

更新日期：1999-09-01 00:00:00

abstract：:C-C chemokine receptor type 5 (CCR5) is a major co-receptor for the entry of human immunodeficiency virus type-1 (HIV-1) into target cells. Human hematopoietic stem cells (hHSCs) with naturally occurring CCR5 deletions (Δ32) or artificially disrupted CCR5 have shown potential for curing acquired immunodeficiency syndr...

journal_title：Human gene therapy

authors： Yao Y,Nashun B,Zhou T,Qin L,Qin L,Zhao S,Xu J,Esteban MA,Chen X

更新日期：2012-02-01 00:00:00