Gene therapy progress and prospects: RNA aptamers.

abstract：:The ability to express recombinant genes in the coronary vasculature and the myocardium holds promise for the treatment of a number of acquired and inherited cardiovascular diseases. Previous in vivo gene transfer approaches in the heart have been limited by relatively low efficiencies of gene transduction. In this re...

journal_title：Gene therapy

authors： Barr E,Carroll J,Kalynych AM,Tripathy SK,Kozarsky K,Wilson JM,Leiden JM

更新日期：1994-01-01 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

abstract：:The production of high-titer recombinant adeno-associated virus (rAAV) vector is essential for treatment of genetic diseases affecting the retina and choroid, where anatomical constraints may limit injectable volumes. Problematically, cytotoxicity arising from overexpression of the transgene during vector production f...

journal_title：Gene therapy

authors： Reid CA,Boye SL,Hauswirth WW,Lipinski DM

更新日期：2017-08-01 00:00:00

abstract：:We have compared the efficacy of daily injection of recombinant leptin protein (rh-leptin) with adenovirus-mediated delivery of the murine or human leptin gene (Ad-leptin) for treatment of obesity in the obese (ob/ob) mouse model. We demonstrate an improved correction profile for obesity and associated surrogate marke...

journal_title：Gene therapy

authors： Morsy MA,Gu MC,Zhao JZ,Holder DJ,Rogers IT,Pouch WJ,Motzel SL,Klein HJ,Gupta SK,Liang X,Tota MR,Rosenblum CI,Caskey CT

更新日期：1998-01-01 00:00:00

abstract：:Adenovirus-mediated gene transfer of interferon gamma (AdIFN) elicits rejection of intracerebral Lewis lung carcinoma. In this system, gene transfer into brain parenchymal cells is both necessary and sufficient to generate the antitumor response. Despite persistent parenchymal inflammation and demyelination, wild-type...

journal_title：Gene therapy

authors： Fathallah-Shaykh HM,Kafrouni AI,Zhao LJ,Diaz-Arrastia R,Garcia JA,Frawley WH,Forman J

更新日期：2000-12-01 00:00:00

abstract：:An amendment to this paper has been published and can be accessed via a link at the top of the paper. ...

journal_title：Gene therapy

authors： Lan Y,Tao Y,Wang Y,Ke J,Yang Q,Liu X,Su B,Wu Y,Lin CP,Zhong G

更新日期：2020-08-01 00:00:00

abstract：:Intervertebral disc degeneration (IVDD) is a multi-factorial process characterized by phenotypic and genotypic changes, which leads to low back pain and disability. Prolonged imbalance between anabolism and catabolism in discs alters their composition resulting in progressive loss of proteoglycans and hydration leadin...

journal_title：Gene therapy

authors： Sampara P,Banala RR,Vemuri SK,Av GR,Gpv S

更新日期：2018-04-01 00:00:00

abstract：:Success of gene therapy for diseases affecting the T cell lineage depends on the thymic repopulation by genetically engineered hematopoietic progenitor cells (HPC). Although it has been shown that retrovirally transduced HPC can repopulate the thymus, little information is available on the effect of the culture protoc...

journal_title：Gene therapy

authors： Verhasselt B,Naessens E,De Smedt M,Plum J

更新日期：2000-05-01 00:00:00

abstract：:The umbilical cord provides a rich source of primitive mesenchymal stem cells (human umbilical cord mesenchymal stem cells (HUMSCs)), which have the potential for transplantation-based treatments of Parkinson's Disease (PD). Our pervious study indicated that adenovirus-associated virus-mediated intrastriatal delivery ...

journal_title：Gene therapy

authors： Xiong N,Zhang Z,Huang J,Chen C,Zhang Z,Jia M,Xiong J,Liu X,Wang F,Cao X,Liang Z,Sun S,Lin Z,Wang T

更新日期：2011-04-01 00:00:00

abstract：:The canine is the most important large animal model for testing novel hemophilia A (HA) treatment. It is often necessary to use canine factor VIII (cFIII) gene or protein for the evaluation of HA treatment in the canine model. However, different biological properties between cFVIII and human FVIII (hFVIII) indicated t...

journal_title：Gene therapy

authors： Wang Q,Dong B,Firrman J,Wu W,Roberts S,Moore AR,Liu LS,Chin MP,Diao Y,Kost J,Xiao W

更新日期：2016-07-01 00:00:00

abstract：:Sendai virus (SeV) is able to transfect airway epithelial cells efficiently in vivo. However, as with other viral vectors, repeated administration leads to reduced gene expression. We have investigated the impact of inducing immunological tolerance to immunodominant T-cell epitopes on gene expression following repeate...

journal_title：Gene therapy

authors： Griesenbach U,Boyton RJ,Somerton L,Garcia SE,Ferrari S,Owaki T,Ya-Fen Z,Geddes DM,Hasegawa M,Altmann DM,Alton EW

更新日期：2006-03-01 00:00:00

abstract：:Therapeutic RNA interference (RNAi) has emerged as a promising approach for the treatment of many incurable diseases, including cancer, infectious disease or neurodegenerative disorders. Demonstration of efficacy and safety in animal models is necessary before planning human application. Our group and others have prev...

journal_title：Gene therapy

authors： Martin JN,Wolken N,Brown T,Dauer WT,Ehrlich ME,Gonzalez-Alegre P

更新日期：2011-07-01 00:00:00

abstract：:Studies reviewed show that lentiviral gene therapy directed either at inhibiting the synthesis of brain acetaldehyde generated from ethanol or at degrading brain acetaldehyde fully prevent ethanol intake by rats bred for their high alcohol preference. However, after animals have chronically consumed alcohol, the above...

journal_title：Gene therapy

authors： Yedy Israel,Quintanilla ME,Ezquer F,Morales P,Rivera-Meza M,Karahanian E,Ezquer M,Herrera-Marschitz M

更新日期：2019-11-01 00:00:00

abstract：:Genetic engineering of T cells for adoptive immunotherapy in cancer patients has shown significant promise. To ensure optimal antitumor activity and safety, the simultaneous expression of multiple genes is frequently required, and short viral-derived 2A sequences are increasingly preferred for this purpose. Concerns e...

journal_title：Gene therapy

authors： Arber C,Abhyankar H,Heslop HE,Brenner MK,Liu H,Dotti G,Savoldo B

更新日期：2013-09-01 00:00:00

abstract：:To confer adenovirus vectors (AdV), the feature of integration into the host cell genome hybrid vectors were characterized in vitro, which express vectors derived from the prototypic foamy virus (FV) in the backbone of a high-capacity AdV. FVs constitute a subfamily of retroviruses with a distinct replication pathway ...

journal_title：Gene therapy

authors： Picard-Maureau M,Kreppel F,Lindemann D,Juretzek T,Herchenröder O,Rethwilm A,Kochanek S,Heinkelein M

更新日期：2004-04-01 00:00:00

abstract：:We have shown that various forms of oligonucleotides, chimeric RNA-DNA oligonucleotide (RDO) and single-stranded oligodeoxynucleotide (ODN), are capable of chromosomal gene alterations in mammalian cells. Using two ODNs we corrected an inactivating mutation in the tyrosinase gene and introduced an activating mutation ...

journal_title：Gene therapy

authors： Alexeev V,Igoucheva O,Yoon K

更新日期：2002-12-01 00:00:00

abstract：:We created Na(+)/HCO3(-) cotransporter 1 (NBCe1) p.W516* knock-in mice as a model of isolated proximal renal tubular acidosis showing early lethality associated with severe metabolic acidosis to investigate the therapeutic effects of prenatal alkalization or posttranscriptional control 124 (PTC124). NBCe1(W516*/W516*)...

journal_title：Gene therapy

authors： Fang YW,Yang SS,Chau T,Nakamura M,Yamazaki O,Seki G,Yamada H,Hsu HM,Cheng CJ,Lin SH

更新日期：2015-05-01 00:00:00

abstract：:In order to characterize protein targeting signals in polarized postmitotic cortical neurones in vitro, we have developed recombinant and amplicon type vectors derived from herpes simplex virus 1 (HSV1) to transfer genes into these cells. We examined the targeting of both bacterial proteins, which lack specific target...

journal_title：Gene therapy

authors： Lowenstein PR,Bain D,Morrison EE,Preston CM,Clissold P,Fournel S,Epstein A,Castro MG

更新日期：1994-01-01 00:00:00

abstract：:Oncolytic viruses hold much promise as novel therapeutic agents that can be combined with conventional therapeutic modalities. Measles virus (MV) is known to enter cells using the signaling lymphocyte activation molecule (SLAM), which is expressed on cells of the immune system. Although human breast cancer cell lines ...

journal_title：Gene therapy

authors： Sugiyama T,Yoneda M,Kuraishi T,Hattori S,Inoue Y,Sato H,Kai C

更新日期：2013-03-01 00:00:00

abstract：:A recombinant adeno-associated virus (rAAV) vector was used to overexpress the anti-apoptotic Bcl-2-family protein, BCL-w, in rat brain. Three weeks after injecting the vector into cerebral cortex and striatum on one side, temporary focal ischemia was induced by occlusion of the ipsilateral middle cerebral artery for ...

journal_title：Gene therapy

authors： Sun Y,Jin K,Clark KR,Peel A,Mao XO,Chang Q,Simon RP,Greenberg DA

更新日期：2003-01-01 00:00:00

abstract：:For certain gene therapy applications, the simultaneous delivery of multiple genes would allow for novel therapies. In the case of adeno-associated virus (AAV) vectors, the limited packaging capacity greatly restricts current methods of carrying multiple transgene cassettes. To address this issue, a recombinant AAV (r...

journal_title：Gene therapy

authors： Foti SB,Samulski RJ,McCown TJ

更新日期：2009-11-01 00:00:00

abstract：:Mucopolysaccharidosis type IIIA (MPS-IIIA) or Sanfilippo A syndrome is a lysosomal storage genetic disease that results from the deficiency of the N-sulfoglucosamine sulfohydrolase (SGSH) protein, a sulfamidase required for the degradation of heparan sulfate glycosaminoglycans (GAGs). The accumulation of these macromo...

journal_title：Gene therapy

authors： Quiviger M,Arfi A,Mansard D,Delacotte L,Pastor M,Scherman D,Marie C

更新日期：2014-12-01 00:00:00

abstract：:Hypoxia initiates an adaptive physiological response in all organisms and plays a role in the pathogenesis of several human diseases. The hypoxia/HIF-inducible factor-1 (HIF-1) transcription factor mediates transcriptional responses to hypoxia by binding to a cis-acting hypoxia-responsive element (HRE) present within ...

journal_title：Gene therapy

authors： Post DE,Van Meir EG

更新日期：2001-12-01 00:00:00

abstract：:To investigate to what extent myeloablation, graft size, and ex vivo manipulation influence the engraftment and long-term survival of transduced murine hematopoietic cells, groups of C57BL/6J (CD45.2) mice receiving total body irradiation (TBI) (1-9 Gy) or no irradiation were transplanted with either transduced bone m...

journal_title：Gene therapy

authors： Puig T,Kádár E,Limón A,Cancelas JA,Eixarch H,Luquín L,García M,Barquinero J

更新日期：2002-11-01 00:00:00

abstract：:Replication-deficient retroviruses have been successfully utilized as vectors, offering an efficient, stable method of therapeutic gene delivery. Many examples exist proving this mode of integrative gene transfer is both effective and safe in cultured systems and clinical trials. Along with their success, severe side ...

journal_title：Gene therapy

authors： Papayannakos C,Daniel R

更新日期：2013-06-01 00:00:00

abstract：:Rhesus macaque tripartite motif (TRIM)5alpha potently inhibits early stages of human immunodeficiency virus (HIV)-1 replication, while the human orthologue has little effect on this virus. We used PCR-based random mutagenesis to construct a large library of human TRIM5alpha variants containing mutations in the PRYSPRY...

journal_title：Gene therapy

authors： Pham QT,Bouchard A,Grütter MG,Berthoux L

更新日期：2010-07-01 00:00:00

abstract：:Angiogenesis plays a pivotal role in tumor growth, tissue invasion and metastasis. Endostatin is an angiogenesis inhibitor and has been shown to reduce tumor growth in animal models. However, therapy with recombinant endostatin protein was hampered by its short half-life and very-low yield of bioactive protein. We per...

journal_title：Gene therapy

authors： Li HL,Li S,Shao JY,Lin XB,Cao Y,Jiang WQ,Liu RY,Zhao P,Zhu XF,Zeng MS,Guan ZZ,Huang W

更新日期：2008-02-01 00:00:00

abstract：:Development of efficient short-term gene transfer technologies for embryonic stem (ES) cells is urgently needed for various existing and new ES cell-based research strategies. In this study, we present a highly efficient, nonviral non-DNA technology for genetic loading of mouse ES cells based on electroporation of def...

journal_title：Gene therapy

authors： Ponsaerts P,Brown JP,Van den Plas D,Van den Eeden L,Van Bockstaele DR,Jorens PG,Van Tendeloo VF,Merregaert J,Singh PB,Berneman ZN

更新日期：2004-11-01 00:00:00

abstract：:Recombinant Semliki Forest virus (SFV) is an attractive viral vector system owing to its ability to allow high efficiency of viral protein expression. To produce recombinant pseudotyped human immunodeficiency virus type 1 (HIV-1) virions, we designed a chimeric SFV/HIV vector system that contains both the HIV-1 cis- a...

journal_title：Gene therapy

authors： Del Vecchio C,Calistri A,Lombardi G,Celegato M,Biasolo MA,Palù G,Parolin C

更新日期：2009-02-01 00:00:00

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