Inhibition of human immunodeficiency virus type 1 by RNA interference using long-hairpin RNA.

abstract：:A number of genetic disorders are manifested in cutaneous epithelium and gene therapy approaches for treatment of such diseases are being considered. A successful gene therapy protocol requires durable and correctly targeted gene expression within the tissue. The continuous renewal and high levels of compartmentalizat...

journal_title：Gene therapy

authors： Ghazizadeh S,Doumeng C,Taichman LB

更新日期：2002-10-01 00:00:00

abstract：:Citrullinemia type 1 (CTLN1) is an autosomal recessive disorder of metabolism caused by a deficiency of argininosuccinate synthetase. Despite optimal management, CTLN1 patients still suffer from lethal metabolic instability and experience life-threatening episodes of acute hyperammonemia. A murine model of CTLN1 (fold...

journal_title：Gene therapy

authors： Chandler RJ,Tarasenko TN,Cusmano-Ozog K,Sun Q,Sutton VR,Venditti CP,McGuire PJ

更新日期：2013-12-01 00:00:00

abstract：:Viral gene vectors often rely on packaging cell lines, which provide the necessary factors in trans for the formation of virus-like particles. Previously, we reported on a first-generation packaging cell line for gene vectors, which are based on the B-lymphotropic Epstein-Barr virus (EBV), a human gamma-herpesvirus. T...

journal_title：Gene therapy

authors： Hettich E,Janz A,Zeidler R,Pich D,Hellebrand E,Weissflog B,Moosmann A,Hammerschmidt W

更新日期：2006-05-01 00:00:00

abstract：:Sustainable correction of severe human genetic disorders of self-renewing tissues, such as the blistering skin disease junctional epidermolysis bullosa (JEB), is facilitated by stable genomic integration of therapeutic genes into somatic tissue stem cells. While integrating viral vectors can achieve this, they suffer ...

journal_title：Gene therapy

authors： Ortiz-Urda S,Lin Q,Yant SR,Keene D,Kay MA,Khavari PA

更新日期：2003-07-01 00:00:00

abstract：:We investigated the efficiency of activated polyamidoamine dendrimers, a new class of nonviral vectors, to transfect rabbit and human corneas in ex vivo culture. In addition to assessing the expression of a marker gene we have demonstrated that this approach can be used to induce the production of TNF receptor fusion ...

journal_title：Gene therapy

authors： Hudde T,Rayner SA,Comer RM,Weber M,Isaacs JD,Waldmann H,Larkin DF,George AJ

更新日期：1999-05-01 00:00:00

abstract：:The growth and metastasis of solid tumors relies on the activities of polypeptide growth factors to recruit stromal tissue and expand the tumor mass. Pleiotrophin (PTN) is a secreted growth factor with angiogenic activity that has been found to contribute to the growth and metastasis of tumors including melanoma. Here...

journal_title：Gene therapy

authors： Malerczyk C,Schulte AM,Czubayko F,Bellon L,Macejak D,Riegel AT,Wellstein A

更新日期：2005-02-01 00:00:00

abstract：:In view of our recent findings that a truncated form of the envelope (Env) glycoprotein of human immunodeficiency virus type 1 (HIV-1) was efficiently incorporated into MoMLV particles, we studied the generation of Moloney murine leukemia virus (MoMLV)/simian immunodeficiency virus (SIV) pseudotypes. Unlike HIV-1, bot...

journal_title：Gene therapy

authors： Indraccolo S,Minuzzo S,Feroli F,Mammano F,Calderazzo F,Chieco-Bianchi L,Amadori A

更新日期：1998-02-01 00:00:00

abstract：:Intramuscular (i.m.) injection of plasmids followed by electropermeabilization is an efficient process to deliver genes into skeletal myofibers that permits proteins to be produced and secreted at therapeutically relevant levels. To further improve skeletal muscle as a bioreactor, we identified a formulation that elev...

journal_title：Gene therapy

authors： Nicol F,Wong M,MacLaughlin FC,Perrard J,Wilson E,Nordstrom JL,Smith LC

更新日期：2002-10-01 00:00:00

abstract：:Partial resistance of primary mouse hepatocytes to lentiviral (LV) vector transduction poses a challenge for ex vivo gene therapy protocols in models of monogenetic liver disease. We thus sought to optimize ex vivo LV gene transfer while preserving the hepatocyte integrity for subsequent transplantation into recipient...

journal_title：Gene therapy

authors： Rothe M,Rittelmeyer I,Iken M,Rüdrich U,Schambach A,Glage S,Manns MP,Baum C,Bock M,Ott M,Modlich U

更新日期：2012-04-01 00:00:00

abstract：:We recently showed that the human telomerase reverse transcriptase (hTERT) promoter induces tumor-specific Bax gene expression and selectively kills various human cancer cells both in vitro and in xenograft tumors. However, it remains unclear whether the hTERT promoter can be used to induce transgene expression in syn...

journal_title：Gene therapy

authors： Gu J,Andreeff M,Roth JA,Fang B

更新日期：2002-01-01 00:00:00

abstract：:This study was designed to determine whether Coxsackie adenovirus receptor (CAR) and alpha nu beta3/alpha nu beta5 integrin co-receptors are involved in adenovirus gene transfer in the rat cochlea. We find that CAR and integrin co-receptors are expressed in every cell subtype transduced by the adenoviral vector Ad5 De...

journal_title：Gene therapy

authors： Venail F,Wang J,Ruel J,Ballana E,Rebillard G,Eybalin M,Arbones M,Bosch A,Puel JL

更新日期：2007-01-01 00:00:00

abstract：:Recombinant human platelet-derived growth factor-BB (rhPDGF-BB) promotes soft tissue and bone healing, and is Food and Drug Administration-approved for treatment of diabetic ulcers and periodontal defects. The short half-life of topical rhPDGF-BB protein application necessitates bolus, high-dose delivery. Gene therapy...

journal_title：Gene therapy

authors： Plonka AB,Khorsand B,Yu N,Sugai JV,Salem AK,Giannobile WV,Elangovan S

更新日期：2017-01-01 00:00:00

abstract：:Mutations in GJB2, which codes for the gap junction (GJ) protein connexin26 (Cx26), are the most common causes of human nonsyndromic hereditary deafness. We inoculated modified adeno-associated viral (AAV) vectors into the scala media of early postnatal conditional Gjb2 knockout mice to drive exogenous Cx26 expression...

journal_title：Gene therapy

authors： Yu Q,Wang Y,Chang Q,Wang J,Gong S,Li H,Lin X

更新日期：2014-01-01 00:00:00

abstract：:Glucagon-like peptide 1 (GLP-1) is released from neuroendocrine cells in the intestine in the postprandial state and augments glucose-stimulated insulin secretion from pancreatic beta cells. To develop non-beta cells that exhibit physiologically regulated insulin secretion, we coexpressed the GLP-1 receptor and human ...

journal_title：Gene therapy

authors： Wu L,Nicholson W,Wu CY,Xu M,McGaha A,Shiota M,Powers AC

更新日期：2003-09-01 00:00:00

abstract：:Introduction of the herpes simplex virus type 1 thymidine kinase gene into tumor cells, followed by the administration of the antiherpes nucleoside analogue ganciclovir has been demonstrated to be effective in eliminating solid tumors in animals. The success of this combination treatment largely depends on the bystand...

journal_title：Gene therapy

authors： Degrève B,De Clercq E,Balzarini J

更新日期：1999-02-01 00:00:00

abstract：:The potential of gene therapy for treatment of lung disease remains unrealised. Early model systems often resulted in promising efficiency of gene transfer, only to prove irreproducible in the clinic. While problems such as induction of host immune responses and duration of expression also need to be addressed, it is ...

journal_title：Gene therapy

authors： McBride S,Rannie D,Harrison DJ

更新日期：2000-04-01 00:00:00

abstract：:Microwave (MW) energy consists of electric and magnetic fields and is able to penetrate deep into biological materials. We investigated the effect of MW (2450 MHz) irradiation on gene delivery in cultured mouse myoblasts and observed enhanced transgene expression. This effect is, however, highly variable and criticall...

journal_title：Gene therapy

authors： Doran TJ,Lu PJ,Vanier GS,Collins MJ,Wu B,Lu QL

更新日期：2009-01-01 00:00:00

abstract：:Over the last years, RNA interference (RNAi) has become a widely used technique that permits the knock-down, and hence functional analysis, of individual genes in vertebrate cells. However, the high failure rate of the RNA molecules used in RNAi experiments continues to be a problem. In this paper, I describe a set of...

journal_title：Gene therapy

authors： Cullen BR

更新日期：2006-03-01 00:00:00

abstract：:Advanced prostate cancer (PC) still remains incurable. Novel immunogene therapy shows promise as treatment strategy that can target both localized and metastasized PC. In this study, we have developed a PC-specific oncolytic adenovirus (Ad-PL-PPT-E1A) armed with fusion gene of prostate-specific antigen and CD40 ligand...

journal_title：Gene therapy

authors： Yang YF,Xue SY,Lu ZZ,Xiao FJ,Yin Y,Zhang QW,Wu CT,Wang H,Wang LS

更新日期：2014-08-01 00:00:00

abstract：:Human immunodeficiency virus (HIV) infection ultimately leads to the destruction of the CD4+ lymphocyte subset and the onset of AIDS. In recent years, several gene therapy procedures making use of retroviral vectors that selectively target HIV susceptible cells have been proposed in order to interfere with HIV product...

journal_title：Gene therapy

authors： Lodge R,Subbramanian RA,Forget J,Lemay G,Cohen EA

更新日期：1998-05-01 00:00:00

abstract：:Hyperammonemia is less severe in arginase 1 deficiency compared with other urea cycle defects. Affected patients manifest hyperargininemia and infrequent episodes of hyperammonemia. Patients typically suffer from neurological impairment with cortical and pyramidal tract deterioration, spasticity, loss of ambulation, s...

journal_title：Gene therapy

authors： Hu C,Tai DS,Park H,Cantero G,Cantero-Nieto G,Chan E,Yudkoff M,Cederbaum SD,Lipshutz GS

更新日期：2015-02-01 00:00:00

abstract：:Retroviral vectors are effective shuttle systems by introducing therapeutically relevant genes stably into the genome of proliferating cells. The majority of vectors applied for research or clinical applications use neomycin for cell selection and identification. To circumvent the time consuming and potentially toxic ...

journal_title：Gene therapy

authors： Machl AW,Planitzer S,Kubbies M

更新日期：1997-04-01 00:00:00

abstract：:The GM2-gangliosidoses are neurological diseases causing premature death, thus developing effective treatment protocols is urgent. GM2-gangliosidoses result from deficiency of a lysosomal enzyme β-hexosaminidase (Hex) and subsequent accumulation of GM2 gangliosides. Genetic changes in HEXA, encoding the Hex α subunit,...

journal_title：Gene therapy

authors： Ou L,Przybilla MJ,Tăbăran AF,Overn P,O'Sullivan MG,Jiang X,Sidhu R,Kell PJ,Ory DS,Whitley CB

更新日期：2020-05-01 00:00:00

abstract：:Cancer therapies that target a single protein or pathway may be limited by their specificity, thus missing key players that control cellular proliferation and contributing to the failure of the treatment. We propose that approaches to cancer therapy that hit multiple targets would limit the chances of escape. To this ...

journal_title：Gene therapy

authors： Xande JG,Dias AP,Tamura RE,Cruz MC,Brito B,Ferreira RA,Strauss BE,Costanzi-Strauss E

更新日期：2020-02-01 00:00:00

abstract：:Various methods for determining the expression of the beta-galactosidase (beta-gal) gene after retroviral transduction were compared as a means to assess retroviral titre. To allow better comparison, different retroviral vectors were constructed carrying two mutants of the green fluorescent protein and assessed as sen...

journal_title：Gene therapy

authors： Klein D,Indraccolo S,von Rombs K,Amadori A,Salmons B,Günzburg WH

更新日期：1997-11-01 00:00:00

abstract：:Urothelial bladder cancer is the most common malignancy of the urinary tract. Although most cases are initially diagnosed as non-muscle-invasive, more than 80% of patients will develop recurrent or metastatic tumors. No effective therapy exists currently for late-stage metastatic tumors. By intravesical application, l...

journal_title：Gene therapy

authors： Zhang KX,Matsui Y,Lee C,Osamu O,Skinner L,Wang J,So A,Rennie PS,Jia WW

更新日期：2016-05-01 00:00:00

abstract：:Dendritic cell (DC)-based vaccines have been applied clinically in the setting of advanced-stage cancer. To date, the clinical efficacy of these vaccines has been limited, possibly owing to the impairment of transferred DC function in cancer-bearing patients. In this study, we examined the therapeutic efficacy of inte...

journal_title：Gene therapy

authors： Tatsumi T,Takehara T,Yamaguchi S,Sasakawa A,Miyagi T,Jinushi M,Sakamori R,Kohga K,Uemura A,Ohkawa K,Storkus WJ,Hayashi N

更新日期：2007-06-01 00:00:00

abstract：:Ideal methods for human gene therapy will eventually include direct gene transfer to defective tissues in a patient in vivo. Toward that goal, we have used high titer, pseudotyped retroviral vectors expressing genes for the Escherichia coli beta-galactosidase (lacZ) or hepatitis B virus surface antigen (HBsAg) to infe...

journal_title：Gene therapy

authors： Miyanohara A,Yee JK,Bouic K,LaPorte P,Friedmann T

更新日期：1995-03-01 00:00:00

abstract：:Previous studies have demonstrated that antigen-specific tolerance could be induced by lipopolysaccharide (LPS)-stimulated B cells retrovirally transduced with an immunoglobulin-antigen (or epitope-containing peptide) fusion construct. To investigate the mechanism of this gene therapy system, we now adapted this appro...

journal_title：Gene therapy

authors： Song L,Wang J,Wang R,Yu M,Sun Y,Han G,Li Y,Qian J,Scott DW,Kang Y,Soukhareva N,Shen B

更新日期：2004-10-01 00:00:00

abstract：:The proteasome is a multisubunit cytosolic protein complex responsible for degrading cytosolic proteins. Several studies have implicated its involvement in the processing of viral particles used for gene delivery, thereby limiting the efficiency of gene transfer. Peptide-based nonviral gene delivery systems are suffic...

journal_title：Gene therapy

authors： Kim J,Chen CP,Rice KG

更新日期：2005-11-01 00:00:00