Gene therapy progress and prospects: bringing gene therapy into medical practice: the evolution of international ethics and the regulatory environment.

abstract：:The oncotropic phenotypes of several viruses correlate with tumor-associated deficiencies within interferon (IFN) signaling pathways. This observation formed the conceptual basis for developing oncolytic viruses deleted for viral proteins that inhibit the host IFN-dependent antiviral response, such as herpes simplex v...

journal_title：Gene therapy

authors： Sobol PT,Hummel JL,Rodrigues RM,Mossman KL

更新日期：2009-09-01 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：:Gene transfer complexes containing poly-L-lysine (poly-K) and DNA with ligands directed at the serpin enzyme complex receptor (sec-R) deliver reporter genes to receptor-bearing cells in vivo. Expression lasts for about 30 days, when complexes containing long-chain poly-K are used. Extending the duration of expression ...

journal_title：Gene therapy

authors： Ziady AG,Kim J,Colla J,Davis PB

更新日期：2004-09-01 00:00:00

abstract：:Neonatal AAV8-mediated Factor IX (F.IX) gene delivery was applied as a model for exploring mechanisms of tolerance induction during immune ontogeny. Intraperitoneal delivery of AAV8/ Factor IX (hF.IX) during weeks 1-4 of life, over a 20-fold dose range, directed stable hF.IX expression, correction of coagulopathy in F...

journal_title：Gene therapy

authors： Shi Y,Falahati R,Zhang J,Flebbe-Rehwaldt L,Gaensler KM

更新日期：2013-10-01 00:00:00

abstract：:The pseudorabies virus (PRV) is a swine alpha herpes virus that is widely used as a neural tracer because of its marked neurotropism and transneuronal transmissibility (Card et al., 1991, 1992; Strack and Loewy 1990). PRV has been used to retrogradely identify spinal cord and brainstem connections to various periphera...

journal_title：Gene therapy

authors： Jasmin L,Tarczy-Hornoch K,Wang H,Levine JD,Basbaum AI

更新日期：1994-01-01 00:00:00

abstract：:Vectors conjugated with ligands recognized by cell surface receptors are of interest for cystic fibrosis gene therapy since these vectors would allow cell-specific targeting. However, an efficient and specific uptake may be abrogated by a subsequent intracellular trafficking leading to an inefficient gene transfer. Th...

journal_title：Gene therapy

authors： Fajac I,Grosse S,Briand P,Monsigny M

更新日期：2002-06-01 00:00:00

abstract：:Citrullinemia is an autosomal recessive disorder caused by the deficiency of argininosuccinate synthetase (AS). It is characterized by elevated levels of blood citrulline and ammonia, which often results in hyperammonemic coma and early neonatal death in affected children. We have explored the use of adenoviral vector...

journal_title：Gene therapy

authors： Ye X,Whiteman B,Jerebtsova M,Batshaw ML

更新日期：2000-10-01 00:00:00

abstract：:Current tools for the inhibition of microRNA (miR) function are limited to modified antisense oligonucleotides, sponges and decoy RNA molecules and none have been used to understand miR function during development. CRISPR/Cas-mediated deletion of miR sequences within the genome requires multiple chromosomal deletions ...

journal_title：Gene therapy

authors： Cao H,Yu W,Li X,Wang J,Gao S,Holton NE,Eliason S,Sharp T,Amendt BA

更新日期：2016-06-01 00:00:00

abstract：:The number of clinical trials using gene transfer technology, either active or under discussion, is increasing rapidly. However, little information is available describing the regulatory procedures or safety specifications that must be considered before initiation of such trials in Europe. We describe the procedure us...

journal_title：Gene therapy

authors： Caplen NJ,Gao X,Hayes P,Elaswarapu R,Fisher G,Kinrade E,Chakera A,Schorr J,Hughes B,Dorin JR

更新日期：1994-03-01 00:00:00

abstract：:Gene therapy of cystic fibrosis (CF) lung disease needs highly efficient delivery and long-lasting complementation of the CFTR (cystic fibrosis transmembrane conductance regulator) gene into the respiratory epithelium. The development of lentiviral vectors has been a recent advance in the field of gene transfer and th...

journal_title：Gene therapy

authors： Copreni E,Penzo M,Carrabino S,Conese M

更新日期：2004-10-01 00:00:00

abstract：:Recombinant adenoviruses (Ad) are attractive vectors for gene transfer in vitro and in vivo. However, the widely used E1-deleted vectors as well as newer generation vectors contain viral sequences, including transcriptional elements for viral gene expression. These viral regulatory elements can interfere with heterolo...

journal_title：Gene therapy

authors： Steinwaerder DS,Lieber A

更新日期：2000-04-01 00:00:00

abstract：:Recombinant Autographa californica multiple nuclear polyhedrosis viruses (AcMNPV) have recently been shown to transduce mammalian cells in vitro. Since baculoviruses offer many advantages over viruses currently used in gene therapy, we have tested them for in vivo gene transfer by constructing a baculovirus bearing a ...

journal_title：Gene therapy

authors： Airenne KJ,Hiltunen MO,Turunen MP,Turunen AM,Laitinen OH,Kulomaa MS,Ylä-Herttuala S

更新日期：2000-09-01 00:00:00

abstract：:Although systemic administration of neutralizing anti-TNF antibodies has been used successfully in treating rheumatoid arthritis, there is a potential for side effects. We transduced a collagen reactive T-cell hybridoma with tissue-specific homing properties to assess therapeutic effects of local delivery to inflamed ...

journal_title：Gene therapy

authors： Smith R,Tarner IH,Hollenhorst M,Lin C,Levicnik AU,Fathman CG,Nolan GP

更新日期：2003-08-01 00:00:00

abstract：:Newly isolated serotypes of AAV readily cross the endothelial barrier to provide efficient transgene delivery throughout the body. However, tissue-specific expression is preferred in most experimental studies and gene therapy protocols. Previous efforts to restrict gene expression to the myocardium often relied on dir...

journal_title：Gene therapy

authors： Prasad KM,Xu Y,Yang Z,Acton ST,French BA

更新日期：2011-01-01 00:00:00

abstract：:As clinical gene therapy has progressed toward realizing its potential, concern over misuse of the technology to enhance performance in athletes is growing. Although 'gene doping' is banned by the World Anti-Doping Agency, its detection remains a major challenge. In this study, we developed a methodology for direct de...

journal_title：Gene therapy

authors： Baoutina A,Coldham T,Bains GS,Emslie KR

更新日期：2010-08-01 00:00:00

abstract：:T-cell dysfunction is thought to be central to the immunodeficiency state seen in patients with the Wiskott-Aldrich syndrome (WAS). Aspects of the WAS phenotype have been corrected in other cell types on introduction of the normal WAS protein (WASP), but the potential for correction of the T-cell defects has not been ...

journal_title：Gene therapy

authors： Strom TS,Gabbard W,Kelly PF,Cunningham JM,Nienhuis AW

更新日期：2003-05-01 00:00:00

abstract：:Inhibition of virus replication by means of RNA interference has been reported for several important human pathogens, including human immunodeficiency virus type 1 (HIV-1). RNA interference against these pathogens has been accomplished by introduction of virus-specific synthetic small interfering RNAs (siRNAs) or DNA ...

journal_title：Gene therapy

authors： Konstantinova P,de Vries W,Haasnoot J,ter Brake O,de Haan P,Berkhout B

更新日期：2006-10-01 00:00:00

abstract：:Complexes of DNA and cationic lipid offer potential advantages for gene transfer to airway epithelia. However, we found that application of DNA-lipid (DMRIE-DOPE) complexes to primary cultures of human ciliated airway epithelia or explants of rabbit trachea generated only low levels of gene transfer. In contrast, when...

journal_title：Gene therapy

authors： Fasbender A,Zabner J,Zeiher BG,Welsh MJ

更新日期：1997-11-01 00:00:00

abstract：:The majority of immunotherapy-based gene therapy protocols consist of ex vivo gene transfer in tumor cells. To prevent further in vivo growth, modified cells must be irradiated before reinjection into patients. The present study examines the effects of gamma-irradiation on transgene expression in transduced leukemic c...

journal_title：Gene therapy

authors： Vereecque R,Saudemont A,Wickham TJ,Gonzalez R,Hetuin D,Fenaux P,Quesnel B

更新日期：2003-02-01 00:00:00

abstract：:Soluble receptors to vascular endothelial growth factor (VEGF) can inhibit its angiogenic effect. Since angiogenesis is involved in wound repair, we hypothesized that adenovirus-mediated gene transfer of a soluble form of VEGF receptor 2 (Flk-1) would attenuate wound healing in mice. C57Bl/6J and genetically diabetic ...

journal_title：Gene therapy

authors： Jacobi J,Tam BY,Sundram U,von Degenfeld G,Blau HM,Kuo CJ,Cooke JP

更新日期：2004-02-01 00:00:00

abstract：:betaTC-tet cells are conditionally immortalized pancreatic beta cells which can confer long-term correction of hyperglycemia when transplanted in syngeneic streptozocin diabetic mice. The use of these cells for control of type I diabetes in humans will require their encapsulation and transplantation in non-native site...

journal_title：Gene therapy

authors： Dupraz P,Rinsch C,Pralong WF,Rolland E,Zufferey R,Trono D,Thorens B

更新日期：1999-06-01 00:00:00

abstract：:Recently, there has been an increasing level of interest in electroporation for gene delivery due to the site-specific nature of the delivery, as well as the high efficiency of the method. Electroporation involves the application of a pulsed electric field to cells to enhance cell permeability, resulting in the transi...

journal_title：Gene therapy

authors： Liu F,Huang L

更新日期：2002-08-01 00:00:00

abstract：:Intravenous delivery of adenoviruses is the optimal route for many gene therapy applications. Once in the blood, coagulation factor X (FX) binds to the adenovirus capsid and protects the virion from natural antibody and classical complement-mediated neutralisation in mice. However, to date, no studies have examined th...

journal_title：Gene therapy

authors： Duffy MR,Doszpoly A,Turner G,Nicklin SA,Baker AH

更新日期：2016-07-01 00:00:00

abstract：:A novel synthetic polypeptide designed as a DNA binding-molecule for liver-specific, receptor-mediated, gene transfer was used to selectively introduce reporter genes into liver cells in the form of plasmid DNA-ligand complexes. The polypeptide was a D-lysine/D-serine copolymer (Lys/Ser = 33/36 or 53/60) modified with...

journal_title：Gene therapy

authors： Hisayasu S,Miyauchi M,Akiyama K,Gotoh T,Satoh S,Shimada T

更新日期：1999-04-01 00:00:00

abstract：:Introducing exogenous genes into cells is one of the most important molecular techniques to study gene functions. Comparing to other type of cells, neurons are more difficult to transfect with cDNAs because they are very sensitive to microenvironmental changes. Among various gene transfer techniques, the Ca(2+)-phosph...

journal_title：Gene therapy

authors： Jiang M,Deng L,Chen G

更新日期：2004-09-01 00:00:00

abstract：:We introduce a lung inflation-fixation protocol to examine the distribution and gene transfer efficiency of fluorescently tagged lipoplexes using fluorescence confocal microscopy within thick lung tissue sections. Using this technique, we tested the hypothesis that factors related to lipoplex distribution were the pre...

journal_title：Gene therapy

authors： Uyechi LS,Gagné L,Thurston G,Szoka FC Jr

更新日期：2001-06-01 00:00:00

abstract：:Plasma apolipoprotein AI (apoAI) and lecithin-cholesterol acyltransferase (LCAT) play important roles in reverse cholesterol transport, promoting the removal of excess cholesterol from peripheral cells and reducing formation of atherosclerotic lesions. Gene augmentation of either apoAI or LCAT, or both, are thus attra...

journal_title：Gene therapy

authors： Fan L,Drew J,Dunckley MG,Owen JS,Dickson G

更新日期：1998-10-01 00:00:00

abstract：:The tumor-suppressor gene PTEN encodes a multifunctional phosphatase that is mutated in a variety of human cancers. PTEN inhibits the phosphatidylinositol 3-kinase pathway and downstream functions, including activation of Akt/protein kinase B (PKB), cell survival, and cell proliferation in tumor cells carrying mutant-...

journal_title：Gene therapy

authors： Saito Y,Swanson X,Mhashilkar AM,Oida Y,Schrock R,Branch CD,Chada S,Zumstein L,Ramesh R

更新日期：2003-11-01 00:00:00

abstract：:Polynucleotide immunization has been employed as a means of inducing immune responses through the introduction of antigen-encoding DNA. While immunization against specific tumor antigens may be achieved through this strategy, various candidate tumor antigens may not be approached via DNA-based vaccines as they represe...

journal_title：Gene therapy

authors： Strong TV,Hampton TA,Louro I,Bilbao G,Conry RM,Curiel DT

更新日期：1997-06-01 00:00:00

abstract：:Current experimental gene therapy approaches for Parkinson's disease (PD) and dementia with Lewy bodies (DLB) include the use of viral vectors expressing antiapoptosis genes, neurotrophic factors and dopaminergic system enzymes. However, since increasing evidence favors a role for alpha-synuclein accumulation in the p...

journal_title：Gene therapy

authors： Hashimoto M,Rockenstein E,Mante M,Crews L,Bar-On P,Gage FH,Marr R,Masliah E

更新日期：2004-12-01 00:00:00