Multidrug resistance 1 gene transfer can confer chemoprotection to human peripheral blood progenitor cells engrafted in immunodeficient mice.

abstract：:Aerosol delivery of adenoviral vectors is of particular interest in regard to gene therapy for cystic fibrosis (CF), with potential advantages of more uniform respiratory delivery, a less invasive approach, and ease of repetition. The AdHCMVsp1LacZ (AdLacZ) adenoviral vector was used to evaluate the feasibility of aer...

journal_title：Human gene therapy

authors： Katkin JP,Gilbert BE,Langston C,French K,Beaudet AL

更新日期：1995-08-01 00:00:00

abstract：:The in vitro genetic manipulation of dendritic cells (DCs) for the expression of foreign proteins or peptides will assist in the development of immunotherapeutic approaches to treat cancer, immunological disorders, and/or infectious diseases. Reports have shown the expansion and differentiation of CD34(+) progenitor c...

journal_title：Human gene therapy

authors： Evans JT,Cravens P,Lipsky PE,Garcia JV

更新日期：2000-12-10 00:00:00

abstract：:Recombinant adeno-associated virus (rAAV) is a prototypical gene therapy vector characterized by excellent safety profiles, wide host range, and the ability to transduce differentiated cells. Numerous rAAV-based vectors providing efficient and sustained expression of transgenes in target tissues have been developed fo...

journal_title：Human gene therapy

authors： Zolotukhin S

更新日期：2005-05-01 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：:Transfer of a herpes simplex virus-derived thymidine kinase (HSV-tk) gene into brain tumor cells and subsequent ganciclovir (GCV) treatment has been shown by others to be an effective treatment in rats with intracerebrally inoculated 9L gliosarcomas. Mechanism of action and reproducibility are, however, still a matter...

journal_title：Human gene therapy

authors： Vincent AJ,Vogels R,Someren GV,Esandi MC,Noteboom JL,Avezaat CJ,Vecht C,Bekkum DW,Valerio D,Bout A,Hoogerbrugge PM

更新日期：1996-01-20 00:00:00

abstract：:Adenoviruses (Ads) have shown great utility as vectors for the delivery of genes to mammalian cells, partly because of their ability to infect a wide range of different cell types independent of the replicative state of the cell. However, Ads do not transduce mature muscle efficiently because of low levels of the natu...

journal_title：Human gene therapy

authors： Bramson JL,Grinshtein N,Meulenbroek RA,Lunde J,Kottachchi D,Lorimer IA,Jasmin BJ,Parks RJ

更新日期：2004-02-01 00:00:00

abstract：:Restoration of correct splicing of βIVS2-654-globin pre-mRNA was previously accomplished in erythroid cells from β-thalassemia/HbE patients by an engineered U7 small nuclear RNA (snRNA) that carried a sequence targeted to the cryptic branch point and an exonic splicing enhancer, U7.BP+623 snRNA. In this study, this ap...

journal_title：Human gene therapy

authors： d'Arqom A,Nualkaew T,Jearawiriyapaisarn N,Kole R,Svasti S

更新日期：2020-11-02 00:00:00

abstract：:Overexpression of human manganese-containing superoxide dismutase (MnSOD) activity has been demonstrated to suppress malignancy in human melanoma and breast carcinoma cells in vitro and in vivo. To study its effects on human oral squamous carcinoma cells, stable transfection and expression of MnSOD in SCC-25 cells hav...

journal_title：Human gene therapy

authors： Liu R,Oberley TD,Oberley LW

更新日期：1997-03-20 00:00:00

abstract：:Sphingosine kinase 1 (SPK1) has been identified as a central mediator of ischemia preconditioning and plays a protective role in ischemia/reperfusion (I/R)-induced cardiomyocyte death. In the present study, we investigated the protective effect of adenovirus-mediated SPK1 gene (Ad-SPK1) transfer on I/R-induced cardiac...

journal_title：Human gene therapy

authors： Duan HF,Wang H,Yi J,Liu HJ,Zhang QW,Li LB,Zhang T,Lu Y,Wu CT,Wang LS

更新日期：2007-11-01 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：:Accurate quantification of gene transfer (or gene correction) is a universal challenge in the field of gene therapy. In developing a clinical trial of lymphocyte gene therapy for Hunter syndrome (mucopolysaccharidosis type II), methods using Southern blot or automated DNA sequencing technology were employed, but found...

journal_title：Human gene therapy

authors： Becker K,Pan D,Whitley CB

更新日期：1999-10-10 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：:In a model of growth-restricted sheep pregnancy, it was previously demonstrated that transient uterine artery VEGF overexpression can improve fetal growth. This approach was tested in guinea-pig pregnancies, where placental physiology is more similar to humans. Fetal growth restriction (FGR) was attained through peri-...

journal_title：Human gene therapy

authors： Swanson AM,Rossi CA,Ofir K,Mehta V,Boyd M,Barker H,Ledwozyw A,Vaughan O,Martin J,Zachary I,Sebire N,Peebles DM,David AL

更新日期：2016-12-01 00:00:00

abstract：:CTL lines directed against HIV-1 antigens were generated from infected individuals and were transduced by the HMB-K(b)HuIFNbeta vector, resulting in low, constitutive expression of interferon beta (IFN-beta). The IFN-beta-transduced cells showed markedly increased HIV-1-specific, MHC class I-restricted CTL activity ag...

journal_title：Human gene therapy

authors： Hadida F,De Maeyer E,Cremer I,Autran B,Baggiolini M,Debré P,Vieillard V

更新日期：1999-07-20 00:00:00

abstract：:Reporter gene-based molecular imaging can provide invaluable information on the fate of cellular therapies postimplantation. Integrating lentiviral vectors (ILVs) are commonly used for stably engineering cells; however, their potential for insertional mutagenesis poses a significant safety concern and barrier to wides...

journal_title：Human gene therapy

authors： Hamilton AM,Foster PJ,Ronald JA

更新日期：2018-10-01 00:00:00

abstract：:Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene that result in the absence of functional protein. In the majority of cases these are out-of-frame deletions that disrupt the reading frame. Several attempts have been made to restore the dystrophin mRNA reading frame by modulation of pre-m...

journal_title：Human gene therapy

authors： Arechavala-Gomeza V,Graham IR,Popplewell LJ,Adams AM,Aartsma-Rus A,Kinali M,Morgan JE,van Deutekom JC,Wilton SD,Dickson G,Muntoni F

更新日期：2007-09-01 00:00:00

abstract：:Somatic gene therapy using nonautologous recombinant cells immunologically protected with alginate microcapsules has been successfully used to treat rodent genetic diseases. We now report the delivery of recombinant gene products to the brain in rodents by implanting microencapsulated cells for the purpose of eventual...

journal_title：Human gene therapy

authors： Ross CJ,Ralph M,Chang PL

更新日期：1999-01-01 00:00:00

abstract：:Adenovirus-polylysine-DNA complexes were evaluated for their capacity to accomplish direct in vivo gene transfer to airway epithelium employing a rodent model. Binary complexes containing transferrin or adenovirus, or combination complexes containing both transferrin and adenovirus, were evaluated. The highest in vitr...

journal_title：Human gene therapy

authors： Gao L,Wagner E,Cotten M,Agarwal S,Harris C,Rømer M,Miller L,Hu PC,Curiel D

更新日期：1993-02-01 00:00:00

abstract：:Mucopolysaccharidosis type IIIA (MPSIIIA) is a rare lysosomal storage disorder caused by mutations in the sulfamidase gene. Accumulation of glycosaminoglycan (GAG) inside the lysosomes is associated with severe neurodegeneration as well as peripheral organ pathological changes leading to death of affected individuals ...

journal_title：Human gene therapy

authors： Ruzo A,Marcó S,García M,Villacampa P,Ribera A,Ayuso E,Maggioni L,Mingozzi F,Haurigot V,Bosch F

更新日期：2012-12-01 00:00:00

abstract：:A phase I clinical trial was conducted in which recombinant adenovirus containing the cystic fibrosis trans-membrane regulator (CFTR) (Ad2/CFTR) was administered by bronchoscopic instillation or aerosolization to the lungs of cystic fibrosis (CF) patients. In this paper, we evaluate the efficiency of Ad2/CFTR-mediated...

journal_title：Human gene therapy

authors： Perricone MA,Morris JE,Pavelka K,Plog MS,O'Sullivan BP,Joseph PM,Dorkin H,Lapey A,Balfour R,Meeker DP,Smith AE,Wadsworth SC,St George JA

更新日期：2001-07-20 00:00:00

abstract：:The combination of immunization strategies with gene therapy methods constitutes a powerful tool for the purpose of genetic immunization. The cutaneous microenvironment, rich in professional antigen-presenting cells and accessory cells capable of initiating and controlling the intensity of specific immune responses, m...

journal_title：Human gene therapy

authors： Larregina AT,Falo LD Jr

更新日期：2000-11-01 00:00:00

abstract：:Urocortin-2 (UCn2) peptide infusion increases cardiac function in patients with heart failure, but chronic peptide infusion is cumbersome, is costly, and provides only short-term benefits. Gene transfer would circumvent these shortcomings. We previously showed that a single intravenous (IV) injection of AAV8.UCn2 incr...

journal_title：Human gene therapy

authors： Lai NC,Gao MH,Giamouridis D,Suarez J,Miyanohara A,Parikh J,Hightower S,Guo T,Dillmann W,Kim YC,Diaz-Juarez J,Hammond HK

更新日期：2015-06-01 00:00:00

abstract：:Classical gene therapy for cystic fibrosis has had limited success because of immune response against viral vectors and short-term expression of cDNA-based transgenes. These limitations could be overcome by delivering the complete genomic CFTR gene on nonintegrating human artificial chromosomes (HACs). Here, we report...

journal_title：Human gene therapy

authors： Rocchi L,Braz C,Cattani S,Ramalho A,Christan S,Edlinger M,Ascenzioni F,Laner A,Kraner S,Amaral M,Schindelhauer D

更新日期：2010-09-01 00:00:00

abstract：:Gene transfer for therapeutic angiogenesis represents a novel treatment for patients with chronic angina refractory to standard medical therapy and not amenable to conventional revascularization. We sought to assess the role of intraoperative multiplane transesophageal echocardiography (MPTEE) in guiding injection of ...

journal_title：Human gene therapy

authors： Esakof DD,Maysky M,Losordo DW,Vale PR,Lathi K,Pastore JO,Symes JF,Isner JM

更新日期：1999-09-20 00:00:00

abstract：:This study focuses on the design, construction, and evaluation of a chimeric promoter for gene therapy applications where it is desirable to have low-level basal expression of the newly transferred gene, which can be induced to higher levels of expression by the administration of pharmacologic agents that can be safel...

journal_title：Human gene therapy

authors： Suzuki M,Singh RN,Crystal RG

更新日期：1996-10-01 00:00:00

abstract：:Oncolytic viruses (OV) are novel cancer gene therapies that are moving toward the forefront of modern medicines. However, their full therapeutic potential is hindered by the lack of convenient and reliable strategies to visualize and quantify OV growth kinetics and therapeutic efficacy in live cells. Here, we present ...

journal_title：Human gene therapy

authors： Quillien L,Top S,Kappler S,Redouté A,Dusetti N,Quentin-Froignant C,Lulka H,Camus C,Buscail L,Gallardo F,Bertagnoli S,Cordelier P

更新日期：2021-01-27 00:00:00

abstract：:At present there is no known effective pharmacological therapy for acute lung injury (ALI). Because keratinocyte growth factor (KGF) promotes epithelial cell growth, intratracheal administration of KGF has the possibility of restoring lung tissue integrity in injured lungs and improving patient outcomes. However, trea...

journal_title：Human gene therapy

authors： Baba Y,Yazawa T,Kanegae Y,Sakamoto S,Saito I,Morimura N,Goto T,Yamada Y,Kurahashi K

更新日期：2007-02-01 00:00:00

abstract：:Retinal ganglion cells (RGCs) play a key role in the pathogenesis and development of glaucoma. The present study aims to investigate the underlying mechanism of long noncoding RNA growth arrest-specific transcript 5 (GAS5) in glaucoma development through regulating the apoptosis of RGCs. Rat models of chronic glaucoma...

journal_title：Human gene therapy

authors： Zhou RR,Li HB,You QS,Rong R,You ML,Xiong K,Huang JF,Xia XB,Ji D

更新日期：2019-12-01 00:00:00

abstract：:Aberrant JAK/STAT3 pathway has been reported to be related to hepatocellular carcinoma (HCC) in many cell lines. In this study, a double-regulated oncolytic adenovirus vector that can replicate and induce a cytopathic effect in alpha-fetoprotein (AFP)-positive HCC cell lines with p53 dysfunction was successfully const...

journal_title：Human gene therapy

authors： Wei RC,Cao X,Gui JH,Zhou XM,Zhong D,Yan QL,Huang WD,Qian QJ,Zhao FL,Liu XY

更新日期：2011-09-01 00:00:00

abstract：:Recent advances in genome sequencing have greatly improved our ability to understand and identify the causes of genetic diseases. However, there remains an urgent need for innovative, safe, and effective treatments for these diseases. CRISPR-based genome editing systems have become important and powerful tools in the ...

journal_title：Human gene therapy

authors： Carlaw TM,Zhang LH,Ross CJD

更新日期：2020-08-01 00:00:00