Prolonged in vivo gene silencing by electroporation-mediated plasmid delivery of small interfering RNA.

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：:Heme oxygenase 1 (HO-1) is an inducible enzyme that catalyzes heme to generate bilirubin, ferritin, and carbon monoxide. Because enhanced expression of HO-1 confers protection against many types of cell and tissue damage by modulating apoptotic cell death or cytokine expression profiles, we hypothesized that adenoviru...

journal_title：Human gene therapy

authors： Tsuburai T,Suzuki M,Nagashima Y,Suzuki S,Inoue S,Hasiba T,Ueda A,Ikehara K,Matsuse T,Ishigatsubo Y

更新日期：2002-11-01 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 first report of in vivo gene delivery to the retina dates back to 1987 when a retroviral vector was injected intraocularly in newborn mice. Later came the observation that retinal cells could be successfully transduced using adenoviral and then adeno-associated and lentiviral vectors. By 2000, it had become clear ...

journal_title：Human gene therapy

authors： Auricchio A,Smith AJ,Ali RR

更新日期：2017-11-01 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：:A human immunodeficiency virus type 1 (HIV-1)-based retroviral vector pseudotyped with HIV envelope containing the herpes simplex virus-thymidine kinase (HSV-TK) gene under the control of the HIV LTR promoter (pHXTKN) was constructed and stably transferred into human CD4(+) H9, CEM, and U937 cells. RNase protection as...

journal_title：Human gene therapy

authors： Miyake K,Iijima O,Suzuki N,Matsukura M,Shimada T

更新日期：2001-02-10 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：:Artemis is a hairpin-opening endonuclease involved in nonhomologous end-joining and V(D)J recombination. Deficiency of Artemis results in radiation-sensitive severe combined immunodeficiency (SCID) characterized by complete absence of T and B cells due to an arrest at the receptor recombination stage. We have generate...

journal_title：Human gene therapy

authors： Multhaup M,Karlen AD,Swanson DL,Wilber A,Somia NV,Cowan MJ,McIvor RS

更新日期：2010-07-01 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：:A major obstacle for the efficacy of cancer gene therapy is the need to transduce a high proportion of tumor cells with genes that directly or indirectly cause their death. During the formation of certain organs, cells compete among themselves to colonize the whole tissue. We reasoned that cell competition could be us...

journal_title：Human gene therapy

authors： Martinez-Quintanilla J,Cascallo M,Fillat C,Alemany R

更新日期：2009-07-01 00:00:00

abstract：:Twenty-eight patients with advanced neovascular age-related macular degeneration (AMD) were given a single intravitreous injection of an E1-, partial E3-, E4-deleted adenoviral vector expressing human pigment epithelium- derived factor (AdPEDF.11). Doses ranging from 10(6) to 10(9.5) particle units (PU) were investiga...

journal_title：Human gene therapy

authors： Campochiaro PA,Nguyen QD,Shah SM,Klein ML,Holz E,Frank RN,Saperstein DA,Gupta A,Stout JT,Macko J,DiBartolomeo R,Wei LL

更新日期：2006-02-01 00:00:00

abstract：:We previously demonstrated that intramuscular plasmid injection serves as a useful method of long-term systemic delivery of cytokines. In the present study, we assess intramuscular DNA injection as a means of systemically delivering interleukin 10 (IL-10), a cytokine with immunosuppressive properties, and preventing t...

journal_title：Human gene therapy

authors： Nitta Y,Tashiro F,Tokui M,Shimada A,Takei I,Tabayashi K,Miyazaki J

更新日期：1998-08-10 00:00:00

abstract：:The enzyme hypoxanthine-guanine phosphoribosyltransferase (HGPRT) expressed by the parasite Trypanosoma brucei (Tb) can convert allopurinol, a purine analogue, to corresponding nucleotides with greater efficiency than its human homologue. We have developed a retroviral system that expresses the parasitic enzyme and te...

journal_title：Human gene therapy

authors： Trudeau C,Yuan S,Galipeau J,Benlimame N,Alaoui-Jamali MA,Batist G

更新日期：2001-09-01 00:00:00

abstract：:Diabetes mellitus is associated with increased risk of heart failure. It has been previously demonstrated in mice that a single injection of adeno-associated virus 8 encoding urocortin 2 (AAV8.UCn2) increases glucose disposal in models of insulin resistance and improves the function of the failing heart. The present s...

journal_title：Human gene therapy

authors： Kim YC,Giamouridis D,Lai NC,Guo T,Xia B,Fu Z,Gao MH,Hammond HK

更新日期：2019-06-01 00:00:00

abstract：:Cell encapsulation offers a safe and manufacturable method for the systemic delivery of therapeutic proteins from genetically engineered cells. However, control of dose delivery remains a major issue with regard to clinical application. We generated populations of immortalized murine NIH 3T3 fibroblasts that secrete m...

journal_title：Human gene therapy

authors： Serguera C,Bohl D,Rolland E,Prevost P,Heard JM

更新日期：1999-02-10 00:00:00

abstract：:The immune response against human immunodeficiency virus type-1 (HIV-1) is believed to play a role in controlling the early stages of disease progression. The cellular immune response, in particular cytotoxic T lymphocyte (CTL) activity, may be important for eliminating virally infected cells in HIV-1-infected individ...

journal_title：Human gene therapy

authors： Laube LS,Burrascano M,Dejesus CE,Howard BD,Johnson MA,Lee WT,Lynn AE,Peters G,Ronlov GS,Townsend KS

更新日期：1994-07-01 00:00:00

abstract：:Recombinant vectors based on adeno-associated virus serotype 8 (AAV8) have been successfully used in the clinic and hold great promise for liver-directed gene therapy. Preexisting immunity against AAV8 or the development of antibodies against the therapeutic transgene product might negatively affect the outcomes of ge...

journal_title：Human gene therapy

authors： Ferla R,Claudiani P,Savarese M,Kozarsky K,Parini R,Scarpa M,Donati MA,Sorge G,Hopwood JJ,Parenti G,Fecarotta S,Nigro V,Sivri HS,Van Der Ploeg A,Andria G,Brunetti-Pierri N,Auricchio A

更新日期：2015-03-01 00:00:00

abstract：:The bystander effect is an important part of tumor kill using gene-directed enzyme prodrug therapy (GDEPT). Recently, we have described a novel enzyme prodrug system using bacterial nitroreductase and the prodrug CB1954 (NTR/CB1954). We demonstrate here the presence of a cell-permeable cytotoxic activity in the condit...

journal_title：Human gene therapy

authors： Bridgewater JA,Knox RJ,Pitts JD,Collins MK,Springer CJ

更新日期：1997-04-10 00:00:00

abstract：:The clinical success of suicide gene therapy using herpes simplex virus type 1 thymidine kinase (TK) is largely dependent on the capacity of this enzyme to effectively induce the death of bystander cells. We have shown that fusion of TK to an 11-amino acid peptide from the basic domain of the human immunodeficiency vi...

journal_title：Human gene therapy

authors： Tasciotti E,Giacca M

更新日期：2005-12-01 00:00:00

abstract：:Trans-dominant mutants of human immunodeficiency virus type 1 (HIV-1) Tat and Rev are attractive candidates for use in gene therapy in the treatment of HIV-1 infections because both are essential for viral replication. Retroviral vectors were constructed to allow either Tat-inducible or Tat- and Rev-inducible expressi...

journal_title：Human gene therapy

authors： Liem SE,Ramezani A,Li X,Joshi S

更新日期：1993-10-01 00:00:00

abstract：:Retinal gene therapy based on adeno-associated viral (AAV) vectors is safe and efficient in humans. The low intrinsic DNA transfer capacity of AAV has been expanded by dual vectors where a large expression cassette is split in two halves independently packaged in two AAV vectors. Dual AAV transduction efficiency, howe...

journal_title：Human gene therapy

authors： Maddalena A,Dell'Aquila F,Giovannelli P,Tiberi P,Wanderlingh LG,Montefusco S,Tornabene P,Iodice C,Visconte F,Carissimo A,Medina DL,Castoria G,Auricchio A

更新日期：2018-08-01 00:00:00

abstract：:Gene delivery via murine-based recombinant retroviral vectors is currently widely used in gene therapy clinical trials. The vectors are engineered to be replication defective by replacing the structural and nonstructural genes of a cloned infectious retrovirus with a therapeutic gene of interest. The retroviral partic...

journal_title：Human gene therapy

authors： Martineau D,Klump WM,McCormack JE,DePolo NJ,Kamantigue E,Petrowski M,Hanlon J,Jolly DJ,Mento SJ,Sajjadi N

更新日期：1997-07-01 00:00:00

abstract：:Stereotactic inoculation of a herpes simplex virus (HSV) gene transfer vector into the hippocampus and caudate of rat brain resulted in limited and transient viral replication and the establishment of latency. Virus attenuation was achieved by insertional inactivation of a viral gene, Us3. Insertion of a lacZ reporter...

journal_title：Human gene therapy

authors： Fink DJ,Sternberg LR,Weber PC,Mata M,Goins WF,Glorioso JC

更新日期：1992-02-01 00:00:00

abstract：:This is an erratum of the published paper "Preclinical Evaluation of Chimeric Antigen Receptor-Modified T Cells Specific to Epithelial Cell Adhesion Molecule for Treating Colorectal Cancer". There are some errors in figure 6C and 7C in the article due to authors' mistakes when preparing the figures. Specifically, repr...

journal_title：Human gene therapy

authors： Wang W

更新日期：2019-08-01 00:00:00

abstract：:Virotherapy is a unique modality for the treatment of cancer with oncolytic viruses (OVs) that selectively infect and lyse tumor cells, spread within tumors, and activate anti-tumor immunity. Various viruses are being developed as OVs preclinically and clinically, several of them engineered to encode therapeutic prote...

journal_title：Human gene therapy

authors： Ungerechts G,Engeland CE,Buchholz CJ,Eberle J,Fechner H,Geletneky K,Holm PS,Kreppel F,Kühnel F,Lang KS,Leber MF,Marchini A,Moehler M,Mühlebach MD,Rommelaere J,Springfeld C,Lauer UM,Nettelbeck DM

更新日期：2017-10-01 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：:Replication-competent adenoviruses may provide a highly efficient means of delivering therapeutic genes to tumors. Previously, we evaluated in vitro a replication-competent adenovirus (Ad5-CD/TKrep) containing a cytosine deaminase (CD)/herpes simplex type 1 thymidine kinase (HSV-1 TK) fusion gene that allows lytic vir...

journal_title：Human gene therapy

authors： Rogulski KR,Wing MS,Paielli DL,Gilbert JD,Kim JH,Freytag SO

更新日期：2000-01-01 00:00:00

abstract：:We evaluated the ability of a replication-deficient, recombinant adenoviral vector to transfer the bifunctional gene GAL-TEK, which expresses a marking/therapeutic gene product, to naturally occurring cat fibrosarcomas in situ. GAL-TEK contains an in-frame fusion of the bacterial LacZ gene for histochemical marking of...

journal_title：Human gene therapy

authors： Marini FC 3rd,Cannon JP,Belmont JW,Shillitoe EJ,Lapeyre JN

更新日期：1995-09-01 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：: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