Pharmacological regulation of the insulin receptor signaling pathway mimics insulin action in cells transduced with viral vectors.

abstract：:Gene therapy for heart diseases requires availability of an efficient vector for gene transfer into myocardium. Recombinant adenovirus expressing the Escherichia coli beta-galactosidase (beta-Gal) gene was shown to infect rat cardiocytes efficiently in vivo. However, a time course of gene expression showed that transg...

journal_title：Human gene therapy

authors： Gilgenkrantz H,Duboc D,Juillard V,Couton D,Pavirani A,Guillet JG,Briand P,Kahn A

更新日期：1995-10-01 00:00:00

abstract：:Targeted therapy produces objective responses in bladder cancer patients, although the responses can be short. Meanwhile, response rates to immune therapy are lower, but the effects are more durable. Based on these findings, it was hypothesized that urothelial carcinoma associated 1 (UCA1)-targeted therapy could syner...

journal_title：Human gene therapy

authors： Zhen S,Lu J,Chen W,Zhao L,Li X

更新日期：2018-12-01 00:00:00

abstract：:Adeno-associated viral (AAV) vectors containing cone-specific promoters have rescued cone photoreceptor function in mouse and dog models of achromatopsia, but cone-specific promoters have not been optimized for use in primates. Using AAV vectors administered by subretinal injection, we evaluated a series of promoters ...

journal_title：Human gene therapy

authors： Ye GJ,Budzynski E,Sonnentag P,Nork TM,Sheibani N,Gurel Z,Boye SL,Peterson JJ,Boye SE,Hauswirth WW,Chulay JD

更新日期：2016-01-01 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：:Isolated methylmalonic acidemia (MMA), a group of autosomal recessive inborn errors of metabolism, is most commonly caused by complete (mut(0)) or partial (mut(-)) deficiency of the enzyme methylmalonyl-CoA mutase (MUT). The severe metabolic instability and increased mortality experienced by many affected individuals,...

journal_title：Human gene therapy

authors： Harrington EA,Sloan JL,Manoli I,Chandler RJ,Schneider M,McGuire PJ,Calcedo R,Wilson JM,Venditti CP

更新日期：2016-05-01 00:00:00

abstract：:Human hematopoietic stem cells (HSCs) are poorly transduced by vectors based on adenovirus serotype 5 (Ad5). This is primarily due to the paucity of the coxsackievirus-Ad receptor on these cells. In an attempt to change the tropism of Ad5, we constructed a series of chimeric E1-deleted Ad5 vectors in which the shaft a...

journal_title：Human gene therapy

authors： Knaän-Shanzer S,Van Der Velde I,Havenga MJ,Lemckert AA,De Vries AA,Valerio D

更新日期：2001-11-01 00:00:00

abstract：:La Crosse virus (LACV)-mediated encephalitis is the most frequently reported arboviral disease in the United States, but to date no vaccine against this virus is available. We have established a new animal model, genetically targeted mice lacking a functional interferon type I receptor (IFNAR-1). These mice show an ag...

journal_title：Human gene therapy

authors： Schuh T,Schultz J,Moelling K,Pavlovic J

更新日期：1999-07-01 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：:Successful gene therapy approaches for metachromatic leukodystrophy (MLD), based either on hematopoietic stem/progenitor cells (HSPCs) or direct central nervous system (CNS) gene transfer, highlighted a requirement for high levels of arylsulfatase A (ARSA) expression to achieve correction of disease manifestations in ...

journal_title：Human gene therapy

authors： Capotondo A,Cesani M,Pepe S,Fasano S,Gregori S,Tononi L,Venneri MA,Brambilla R,Quattrini A,Ballabio A,Cosma MP,Naldini L,Biffi A

更新日期：2007-09-01 00:00:00

abstract：:The purpose of this study was to determine the safety and antitumor activity of IFN-gamma retroviral vector in patients with advanced melanoma. Seventeen patients (9 single courses, 8 multiple courses) received a total of 363 intratumor injections of IFN-gamma retroviral vector (1 x 10(7) PFU/ml administered at 0.3, 0...

journal_title：Human gene therapy

authors： Nemunaitis J,Fong T,Burrows F,Bruce J,Peters G,Ognoskie N,Meyer W,Wynne D,Kerr R,Pippen J,Oldham F,Ando D

更新日期：1999-05-20 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：:The immunogenicity of adenovirus vectors remains a major obstacle to their safe and efficacious use for gene therapy. In order to identify T-cell epitopes directly from adenoviruses, four viral protein sequences were screened for the well-characterized 9-mer HLA-A2 binding motif. Peripheral blood mononuclear cells (PB...

journal_title：Human gene therapy

authors： Olive M,Eisenlohr L,Flomenberg N,Hsu S,Flomenberg P

更新日期：2002-07-01 00:00:00

abstract：:This multicenter phase I/II study evaluated the safety, pharmacokinetics, and antitumor effects of repeated doses of NV1020, a genetically engineered oncolytic herpes simplex virus, in patients with advanced metastatic colorectal cancer (mCRC). Patients with liver-dominant mCRC received four fixed NV1020 doses via wee...

journal_title：Human gene therapy

authors： Geevarghese SK,Geller DA,de Haan HA,Hörer M,Knoll AE,Mescheder A,Nemunaitis J,Reid TR,Sze DY,Tanabe KK,Tawfik H

更新日期：2010-09-01 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：:Malignant mesothelioma is a tumor of the pleura for which there is no satisfactory treatment. It is almost universally fatal, regardless of the stage of the tumor at the time of diagnosis. Current treatment modalities include surgery, chemotherapy, and radiation therapy, although in some series none of these modalitie...

journal_title：Human gene therapy

authors： Schwarzenberger P,Harrison L,Weinacker A,Marrogi A,Byrne P,Ramesh R,Theodossiou C,Gaumer R,Summer W,Freeman SM,Kolls JK

更新日期：1998-11-20 00:00:00

abstract：:Human serum is known to inactivate many retroviruses, including murine leukemia viruses (MLV). Exposure of vectors based on MLV to human serum components would presumably decrease the efficiency of gene transfer in vivo. Human serum also lyses xenogeneic cells, which would affect the survival of retroviral vector pack...

journal_title：Human gene therapy

authors： Russell DW,Berger MS,Miller AD

更新日期：1995-05-01 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：:Unlike oncoretroviruses, lentiviral vectors can insert large genes and can target both dividing and nondividing cells; thus they hold unique promise as gene transfer agents. To enhance target range, the native lentiviral envelope glycoprotein is replaced (pseudotyped) with vesicular stomatitis virus G (VSVG), and the ...

journal_title：Human gene therapy

authors： Kumar M,Bradow BP,Zimmerberg J

更新日期：2003-01-01 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：:If established cultured cell lines genetically modified to secrete desired gene products could be implanted in different allogeneic recipients without immune rejection, novel gene products would be delivered more cost effectively. We tested this strategy by encapsulating mouse Ltk- cells transfected with the human gro...

journal_title：Human gene therapy

authors： Chang PL,Shen N,Westcott AJ

更新日期：1993-08-01 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：:Combining chemotherapy and immunotherapy is problematic because chemotherapy can ablate the immune responses initiated by modulators of the immune system. We hypothesized that protection of immunocompetent cells from the toxic effects of chemotherapy, using drug resistance gene therapy strategies, would allow the comb...

journal_title：Human gene therapy

authors： McMillin DW,Hewes B,Gangadharan B,Archer DR,Mittler RS,Spencer HT

更新日期：2006-08-01 00:00:00

abstract：:Targeted integration into a genomic safe harbor, such as the AAVS1 locus on chromosome 19, promises predictable transgene expression and reduces the risk of insertional mutagenesis in the host genome. The application of gamma-retroviral long terminal repeat (LTR)-driven vectors, which semirandomly integrate into the g...

journal_title：Human gene therapy

authors： Klatt D,Cheng E,Hoffmann D,Santilli G,Thrasher AJ,Brendel C,Schambach A

更新日期：2020-02-01 00:00:00

abstract：:Lentiviral vectors hold great promise for the genetic correction of various inherited diseases. However, lentiviral vector biology is still not completely understood and warrants the precise decoding of molecular mechanisms underlying integration and post-translational modification. This study investigated a series of...

journal_title：Human gene therapy

authors： Scholz SJ,Fronza R,Bartholomä CC,Cesana D,Montini E,von Kalle C,Gil-Farina I,Schmidt M

更新日期：2017-10-01 00:00:00

abstract：:Gene transfer of the herpes simplex virus thymidine kinase (TK) gene associated with ganciclovir (GCV) treatment can lead to death of TK-expressing cells, and of neighboring TK- cells because of the bystander effect. Thus, a small proportion of TK+ cells in a tumor can lead to its complete regression after GCV treatme...

journal_title：Human gene therapy

authors： Qiao J,Black ME,Caruso M

更新日期：2000-07-20 00:00:00

abstract：:We have shown that adenovirus-mediated manipulation of apoptotic genes such as bax could be a therapeutic option for prostate cancer. Unfortunately, the response of experimental prostate tumors to a single therapeutic gene of the apoptotic pathway is short-lived, and most of these tumors relapse after a short period o...

journal_title：Human gene therapy

authors： Zhang Y,Yu J,Unni E,Shao TC,Nan B,Snabboon T,Kasper S,Andriani F,Denner L,Marcelli M

更新日期：2002-11-20 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：: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：: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：:Administration of plasmid/lipid complexes to the lung airways for the treatment of metastatic pulmonary diseases represents a new strategy of gene therapy. In this study we present evidence that intratracheal administration of a plasmid encoding murine IL-12 complexed with N-[1-(2,3-dioleyloxy)propyl)-N,N,N-trimethyla...

journal_title：Human gene therapy

authors： Blezinger P,Freimark BD,Matar M,Wilson E,Singhal A,Min W,Nordstrom JL,Pericle F

更新日期：1999-03-20 00:00:00