Antisense oligonucleotides for the treatment of spinal muscular atrophy.

abstract：:Evaluation of the potential role of dendritic cells (DCs) as adjuvants for tumor vaccination has focused primarily on techniques that load DCs with peptide tumor antigens. Our aim has been to optimize the induction of antitumor immunity by enhancing the ability of DCs to present tumor-associated antigens endogenously ...

journal_title：Human gene therapy

authors： Wan Y,Bramson J,Carter R,Graham F,Gauldie J

更新日期：1997-07-20 00:00:00

abstract：:Adoptive cellular therapy provides the promise of a potentially powerful general treatment for cancer. Although this is a complex and challenging field, there have been major advances in basic and translational research resulting in clinical trial activity that is now beginning to confirm this promise. However, these ...

journal_title：Human gene therapy

authors： Hawkins RE,Gilham DE,Debets R,Eshhar Z,Taylor N,Abken H,Schumacher TN,ATTACK Consortium

更新日期：2010-06-01 00:00:00

abstract：:Systemic administration of adenoviral vectors leads to activation of innate and antigen-specific immunity. In an attempt to diminish T and B cell-specific immune responses to E1-deleted adenoviral vectors, capsid proteins were modified with various activated monomethoxypolyethylene glycols (MPEGs). The impact of this ...

journal_title：Human gene therapy

authors： Croyle MA,Chirmule N,Zhang Y,Wilson JM

更新日期：2002-10-10 00:00:00

abstract：:In previous studies we showed that low-dose irradiation and immunosuppression with cyclosporine and mycophenolate mofetil prolonged in vivo persistence of gene-modified T cells but was unable to induce tolerance. We hypothesized that the lack of sustained antigen presentation because of the limited life span of the in...

journal_title：Human gene therapy

authors： Piasecki JC,Beagles K,Beard BC,Riddell S,Kiem HP

更新日期：2008-01-01 00:00:00

abstract：:Esophageal cancer is characterized by rapid clinical progression and poor prognosis, due to early-stage invasion of adjacent tissues and metastasis. Tissue factor pathway inhibitor-2 (TFPI-2) has been implicated as a metastasis-associated gene in many types of tumors. Here we describe the potential involvement of TFPI...

journal_title：Human gene therapy

authors： Ran Y,Pan J,Hu H,Zhou Z,Sun L,Peng L,Yu L,Sun L,Liu J,Yang Z

更新日期：2009-01-01 00:00:00

abstract：:Gene therapy for hemophilia B has been shown to result in long-term expression and immune tolerance to factor IX (F.IX) after in vivo transduction of hepatocytes with adeno-associated viral (AAV-2) vectors in experimental animals. An optimized protocol was effective in several strains of mice with a factor 9 gene dele...

journal_title：Human gene therapy

authors： Cooper M,Nayak S,Hoffman BE,Terhorst C,Cao O,Herzog RW

更新日期：2009-07-01 00:00:00

abstract：:Autoimmune destruction of islets in the pancreas leads to the development of insulin-dependent diabetes mellitus (IDDM). Replacement of insulin-producing tissue by transplantation of islets provides a cure to disease but requires immunosuppression or a means of controlling anti-graft immune responses. To promote islet...

journal_title：Human gene therapy

authors： Gallichan WS,Kafri T,Krahl T,Verma IM,Sarvetnick N

更新日期：1998-12-10 00:00:00

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：:Targeted delivery of intravenously administered genetically altered cells or stem cells is still in an early stage of investigation. We developed a method of delivering iron oxide (ferumoxide)-labeled mesenchymal stem cells (MSCs) to a targeted area in an animal model by applying an external magnet. Rats with or witho...

journal_title：Human gene therapy

authors： Arbab AS,Jordan EK,Wilson LB,Yocum GT,Lewis BK,Frank JA

更新日期：2004-04-01 00:00:00

abstract：:Mice bearing breast tumors were treated with a single dose of an adenovirus expressing interleukin-12 (AdmIL-12.1) injected intratumorally, which produced regressions in greater than 75% of the treated tumors; approximately one-third of the animals remained tumor free. Complete regression was associated with immunity ...

journal_title：Human gene therapy

authors： Bramson JL,Hitt M,Addison CL,Muller WJ,Gauldie J,Graham FL

更新日期：1996-10-20 00:00:00

abstract：:Adenovirus vectors transduce liver hepatocytes with extreme efficiency; however, transgene expression is eliminated within 2 weeks. Extinction of transgene expression has been attributed to infiltrating cytotoxic T lymphocytes (CTLs) in the liver in a process that resembles a number of human diseases, including viral ...

journal_title：Human gene therapy

authors： Chirmule N,Moscioni AD,Qian Y,Qian R,Chen Y,Wilson JM

更新日期：1999-01-20 00:00:00

abstract：:Immunologically sensitized recipients present one of the most critical problems in clinical organ transplantation today, since preformed antibodies rapidly destroy donor tissue expressing specific MHC class I antigens (Ag). Therefore, sensitized patients are either unable to receive a compatible organ, or experience a...

journal_title：Human gene therapy

authors： Geissler EK,Graeb C,Tange S,Guba M,Jauch KW,Scherer MN

更新日期：2000-02-10 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：:A novel retroviral vector has been designed based on a Friend-murine leukemia virus (Fr-MuLV) FB29 strain. The latter has been selected according to characteristics of pathogenicity in mice where it induces a disease of the haemopoietic system affecting all lineages. Higher infectivity has also been demonstrated as co...

journal_title：Human gene therapy

authors： Cohen-Haguenauer O,Restrepo LM,Masset M,Bayer J,Dal Cortivo L,Marolleau JP,Benbunan M,Boiron M,Marty M

更新日期：1998-01-20 00:00:00

abstract：:Engineered measles virus (MV) strains deriving from the vaccine lineage represent a promising oncolytic platform and are currently being tested in phase I trials. In this study, we have demonstrated that MV strains genetically engineered to express the human sodium iodide symporter (NIS) have significant antitumor act...

journal_title：Human gene therapy

authors： Opyrchal M,Allen C,Iankov I,Aderca I,Schroeder M,Sarkaria J,Galanis E

更新日期：2012-04-01 00:00:00

abstract：:Hereditary tyrosinemia type 1 (HT1) is an autosomal recessive disorder caused by deficiency of fumarylacetoacetate hydrolase (FAH). It has been previously shown that ex vivo hepatocyte-directed gene therapy using an integrating lentiviral vector to replace the defective Fah gene can cure liver disease in small- and la...

journal_title：Human gene therapy

authors： VanLith C,Guthman R,Nicolas CT,Allen K,Du Z,Joo DJ,Nyberg SL,Lillegard JB,Hickey RD

更新日期：2018-11-01 00:00:00

abstract：:Interleukin-12 (IL-12) is a heterodimeric cytokine that plays an important role in the development of cellular immunity. Clinical applications for this lymphokine include resolution of infectious disease, cancer immunotherapy, and boosting cellular immunity in AIDS patients. When using IL-12 and other cytokines therap...

journal_title：Human gene therapy

authors： Bramson J,Hitt M,Gallichan WS,Rosenthal KL,Gauldie J,Graham FL

更新日期：1996-02-10 00:00:00

abstract：:Extracellular vesicles (EVs) being released from two adjacent adeno-associated virus serotype 1 (AAV1)-producing 293T cells are shown by electron microscopy. We have shown that AAV vectors can associate with EVs and enter the media. Furthermore, we have recently reported that EV-associated AAV has robust gene delivery...

journal_title：Human gene therapy

authors： Fitzpatrick Z,György B,Skog J,Maguire CA

更新日期：2014-09-01 00:00:00

abstract：:pZIG(hGCSFR) is a retroviral vector that can co-express two genes and also provides alternative selection markers. This retroviral vector has been constructed to incorporate an internal ribosome entry site (IRES) element to co-express two exogenous genes in mammalian cells. Two marker/selection genes have been cloned ...

journal_title：Human gene therapy

authors： Saleh M

更新日期：1997-05-20 00:00:00

abstract：:Oncolytic measles virus (MV) encoding the human thyroidal sodium iodide symporter (MV-NIS) has proved to be safe after intraperitoneal or intravenous administration in patients with ovarian cancer or multiple myeloma, respectively, but it has not yet been administered through intratumoral injection in humans. Squamous...

journal_title：Human gene therapy

authors： Li H,Peng KW,Russell SJ

更新日期：2012-03-01 00:00:00

abstract：:For the successful application of RNA interference in vivo, it is desired to achieve (local) delivery of small interfering RNAs (siRNAs) and long-term gene silencing. Nonviral electrodelivery is suitable to obtain local and prolonged expression of transgenes. By intramuscular electrodelivery of a plasmid in which two ...

journal_title：Human gene therapy

authors： Eefting D,Grimbergen JM,de Vries MR,van Weel V,Kaijzel EL,Que I,Moon RT,Löwik CW,van Bockel JH,Quax PH

更新日期：2007-09-01 00:00:00

abstract：:DNA vaccination is an attractive approach for tumor immunotherapy because of its stability and simplicity of delivery. Advances demonstrate that helper T cell responses play a critical role in initiating immune responses. The aim of the current study is to test whether targeting HPV-16 E7 to the endosomal/lysosomal co...

journal_title：Human gene therapy

authors： Ji H,Wang TL,Chen CH,Pai SI,Hung CF,Lin KY,Kurman RJ,Pardoll DM,Wu TC

更新日期：1999-11-20 00:00:00

abstract：:Gutted adenoviral (Ad) vectors have a greater cloning capacity and elicit less immune response than conventional Ad vectors. Unfortunately, clinical use of gutted vectors has been slowed by production difficulties, including low yield and a tendency for recombinant virus to emerge. These two problems are related, beca...

journal_title：Human gene therapy

authors： Hartigan-O'Connor D,Barjot C,Crawford R,Chamberlain JS

更新日期：2002-03-01 00:00:00

abstract：:RNA interference (RNAi) is an evolutionarily conserved mechanism of posttranscriptional gene-specific silencing. For in vivo applications, RNAi has been hampered until recently by inefficient delivery methods and by the transient nature of the gene suppression. Lentiviral vectors (LVs) hold great promise for gene ther...

journal_title：Human gene therapy

authors： Van den Haute C,Eggermont K,Nuttin B,Debyser Z,Baekelandt V

更新日期：2003-12-10 00:00:00

abstract：:As an alternative to virus-mediated gene transfer, we previously demonstrated a simple, safe, and efficient transfer of foreign gene into the central nervous system using continuous injection of a plasmid DNA-cationic liposome complex. To explore whether this approach can be applied to the treatment of certain neurolo...

journal_title：Human gene therapy

authors： Imaoka T,Date I,Ohmoto T,Nagatsu T

更新日期：1998-05-01 00:00:00

abstract：:Gene therapy has evolved into a tempting strategy for the management of cancer and other life-threatening diseases. Various approaches employ retroviral vectors to deliver the therapeutic gene. The profound knowledge about retrovirus biology allows the generation of increasingly advanced vector systems as well as an a...

journal_title：Human gene therapy

authors： Brandtner EM,Kodajova P,Knapp E,Ertl R,Tabotta W,Salmons B,Günzburg WH,Hohenadl C

更新日期：2008-01-01 00:00:00

abstract：:The efficient and specific introduction of genes into cancer cells in vivo remains a major challenge for current gene therapy modalities. Peptides possess appropriate properties to serve as tumor-targeting agents. Thus, finding new cancer-selective peptides directing gene transfer to neoplastic cells by reducing trans...

journal_title：Human gene therapy

authors： Böckmann M,Hilken G,Schmidt A,Cranston AN,Tannapfel A,Drosten M,Frilling A,Ponder BA,Pützer BM

更新日期：2005-11-01 00:00:00

abstract：:Blood vessels are among the easiest targets for gene therapy. However, no data are available about the safety and feasibility of intracoronary gene transfer in humans. We studied the safety and efficacy of catheter-mediated vascular endothelial growth factor (VEGF) plasmid/liposome (P/L) gene transfer in human coronar...

journal_title：Human gene therapy

authors： Laitinen M,Hartikainen J,Hiltunen MO,Eränen J,Kiviniemi M,Närvänen O,Mäkinen K,Manninen H,Syvänne M,Martin JF,Laakso M,Ylä-Herttuala S

更新日期：2000-01-20 00:00:00

abstract：:Adrenomedullin (AM) has been shown to protect against ischemia/reperfusion-induced myocardial infarction and apoptosis. In the present study, we examined the potential neuroprotective action of delayed AM gene transfer in cerebral ischemia. Three days after a 1-hr occlusion of the middle cerebral artery (MCAO), rats w...

journal_title：Human gene therapy

authors： Xia CF,Yin H,Borlongan CV,Chao J,Chao L

更新日期：2004-12-01 00:00:00

abstract：:In vivo electroporation of plasmid DNA (DNA-EP) is an efficient and safe method for vaccines. It results in increased DNA uptake, enhances protein expression, and augments immune responses to the target antigen in a variety of species. To further improve the efficacy of DNA-EP, we evaluated small interfering RNA (siRN...

journal_title：Human gene therapy

authors： Dharmapuri S,Aurisicchio L,Biondo A,Welsh N,Ciliberto G,La Monica N

更新日期：2009-06-01 00:00:00