Widespread transduction of astrocytes and neurons in the mouse central nervous system after systemic delivery of a self-complementary AAV-PHP.B vector.

abstract：:The recent surge of DNA sequence information resulting from the efforts of agencies interested in deciphering the human genetic code has facilitated technological developments that have been critical in the identification of genes associated with numerous disease pathologies. In addition, these efforts have opened the...

journal_title：Gene therapy

authors： Gruenert DC,Novelli G,Dallapiccola B,Colosimo A

更新日期：2002-06-01 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：:Gene modification of malignant cells to express immune stimulators (cytokines and immune costimulators) has provided the basis for a novel form of immunotherapy. Using a MPSV-based retroviral vector with hygromycin resistance gene as a selectable marker, we have studied retrovirus-mediated gene transfer of an immune c...

journal_title：Gene therapy

authors： Hirst WJ,Buggins A,Darling D,Gäken J,Farzaneh F,Mufti GJ

更新日期：1997-07-01 00:00:00

abstract：:Human adenovirus (Ad) vectors are extensively used as gene transfer vehicles. However, a serious obstacle for the use of these vectors in clinical applications is due to pre-existing immunity to human Ads affecting the efficacy of gene transfer. One of the approaches to circumvent host immune response could be the dev...

journal_title：Gene therapy

authors： Logunov DY,Ilyinskaya GV,Cherenova LV,Verhovskaya LV,Shmarov MM,Chumakov PM,Kopnin BP,Naroditsky BS

更新日期：2004-01-01 00:00:00

abstract：:Multiple antitumor modalities may be necessary to overcome lung tumor-mediated immunosuppression and effectively treat non-small cell lung cancer (NSCLC). To evaluate a multimodality gene therapy approach for control of local tumor growth, a weakly immunogenic murine alveolar cell carcinoma, L1C2, was transduced with ...

journal_title：Gene therapy

authors： Sharma S,Miller PW,Stolina M,Zhu L,Huang M,Paul RW,Dubinett SM

更新日期：1997-12-01 00:00:00

abstract：:For patients with metastatic prostate cancer, the 5-year survival rate of 31% points to a need for novel therapies and improvement of existing modalities. We propose that p53 gene therapy and chemotherapy, when combined, will provide superior tumor cell killing for the treatment of prostate carcinoma. To this end, we ...

journal_title：Gene therapy

authors： Tamura RE,Lana MG,Costanzi-Strauss E,Strauss BE

更新日期：2020-02-01 00:00:00

abstract：:Epstein-Barr virus (EBV)-associated B-cell lymphoproliferative disease (LPD) after hematopoietic stem cell or solid organ transplantation remains a life-threatening complication. Expression of the virus-encoded gene product, EBER, has been shown to prevent apoptosis via blockade of PKR activation. As PKR is a major ce...

journal_title：Gene therapy

authors： Wang PY,Currier MA,Hansford L,Kaplan D,Chiocca EA,Uchida H,Goins WF,Cohen JB,Glorioso JC,van Kuppevelt TH,Mo X,Cripe TP

更新日期：2013-07-01 00:00:00

abstract：:We explored the possibility of using a genetic approach to inhibit integrin-mediated endothelial cell adhesion and survival. We constructed recombinant adenoviruses (Ads) expressing chimeric proteins consisting of the cytoplasmic and transmembrane domains of integrin beta1 (CH1), beta3 (CH3) or the beta1 transmembrane...

journal_title：Gene therapy

authors： Oguey D,George PW,Rüegg C

更新日期：2000-08-01 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：:Adoptive cell therapy with chimeric antigen receptor (CAR)-modified T cells showed remarkable therapeutic efficacy in the treatment of leukaemia/lymphoma. However, the application to a variety of cancer entities is often constricted by the non-availability of a single chain antibody (scFv), which is usually the target...

journal_title：Gene therapy

authors： Faitschuk E,Nagy V,Hombach AA,Abken H

更新日期：2016-10-01 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：:Efficient transduction of the peripheral nervous system (PNS) is required for gene therapy of acquired and inherited neuropathies, neuromuscular diseases and for pain treatment. We have characterized the tropism and transduction efficiency of different adeno-associated vectors (AAV) pseudotypes after sciatic nerve inj...

journal_title：Gene therapy

authors： Homs J,Ariza L,Pagès G,Udina E,Navarro X,Chillón M,Bosch A

更新日期：2011-06-01 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：:Cultured keratinocyte allografts from unrelated donors can be readily grown as sheets in large-scale cell culture and have been used as an immediate skin cover for severely burned patients. Despite the absence of passenger leukocytes and the unlimited amount of material that can be obtained for permanent skin coverage...

journal_title：Gene therapy

authors： Mhashilkar AM,Doebis C,Seifert M,Busch A,Zani C,Soo Hoo J,Nagy M,Ritter T,Volk HD,Marasco WA

更新日期：2002-03-01 00:00:00

abstract：:Catheter-based percutaneous transluminal gene delivery (PTGD) into the coronary artery still falls behind the expectations of an efficient myocardial gene delivery system. In this study gene delivery was applied by selective pressure-regulated retroinfusion through the coronary veins to prolong adhesion of replication...

journal_title：Gene therapy

authors： Boekstegers P,von Degenfeld G,Giehrl W,Heinrich D,Hullin R,Kupatt C,Steinbeck G,Baretton G,Middeler G,Katus H,Franz WM

更新日期：2000-02-01 00:00:00

abstract：:The concept and application of ethical principles in the context of medical research is changing rapidly. This is especially true for fast moving fields such as gene therapy, a relatively new but rapidly maturing field offering opportunities to influence health at a fundamental level, which may become a cornerstone of...

journal_title：Gene therapy

authors： Spink J,Geddes D

更新日期：2004-11-01 00:00:00

abstract：:This study was designed to evaluate the utility of positron emission tomography (PET) to quantify the magnitude and spatial distribution of transgene expression after different methods of adenoviral vector delivery (with surfactant- and saline-based vehicles) within rat lungs. In all, 17 animals (eight in the surfacta...

journal_title：Gene therapy

authors： Richard JC,Factor P,Welch LC,Schuster DP

更新日期：2003-12-01 00:00:00

abstract：:Glucagon-like peptide-1 (GLP-1) is an incretin hormone that regulates blood glucose level post-prandially. It has been proposed that GLP-1 can be used in type 2 diabetes (T2D) mellitus treatment because of its insulinotropic action. Despite its remarkable advantages, GLP-1 suffers the disadvantage of an extremely shor...

journal_title：Gene therapy

authors： Jean M,Alameh M,Buschmann MD,Merzouki A

更新日期：2011-08-01 00:00:00

abstract：:We evaluated the efficacy of equine infectious anaemia virus (EIAV)-based lentiviral vectors encoding endostatin (EIAV.endostatin) or angiostatin (EIAV.angiostatin) in inhibiting angiogenesis and vascular hyperpermeability in the laser-induced model of choroidal neovascularisation (CNV). Equine infectious anaemia viru...

journal_title：Gene therapy

authors： Balaggan KS,Binley K,Esapa M,MacLaren RE,Iqball S,Duran Y,Pearson RA,Kan O,Barker SE,Smith AJ,Bainbridge JW,Naylor S,Ali RR

更新日期：2006-08-01 00:00:00

abstract：:The expression of naked plasmid DNA coding for firefly luciferase (pRSVluc) or a secreted protein, human-alpha-1-antitrypsin (pRcCMVhAAT) in mouse skeletal muscle was characterized following administration by an improved intramuscular injection technique. Injection guided by intense illumination along the longitudinal...

journal_title：Gene therapy

authors： Levy MY,Barron LG,Meyer KB,Szoka FC Jr

更新日期：1996-03-01 00:00:00

abstract：:A simple, efficient and reproducible method to transduce proteins into mammalian cells has not been established. Here we describe a novel protein transduction method based on a lentiviral vector. We have developed a method to package several thousand foreign protein molecules into a lentivirus-like nanoparticle (LENA)...

journal_title：Gene therapy

authors： Aoki T,Miyauchi K,Urano E,Ichikawa R,Komano J

更新日期：2011-09-01 00:00:00

abstract：:Lentiviral vectors pseudotyped with the baculovirus envelope protein GP64 transduce primary cultures of human airway epithelia (HAE) at their apical surface. Our goal in this study was to harness a directed evolution approach to develop a novel envelope glycoprotein with increased transduction properties for HAE. Usin...

journal_title：Gene therapy

authors： Sinn PL,Hwang BY,Li N,Ortiz JLS,Shirazi E,Parekh KR,Cooney AL,Schaffer DV,McCray PB Jr

更新日期：2017-10-01 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：:Several of the current techniques for transfer of both oligonucleotide and plasmid DNA into the myocardium are impaired by low efficiency and toxicity. To improve gene transfer techniques, especially into the whole heart, a gene transfer method involving liposome in conjunction with a viral envelope (HVJ-liposome) was...

journal_title：Gene therapy

authors： Sawa Y,Kaneda Y,Bai HZ,Suzuki K,Fujimoto J,Morishita R,Matsuda H

更新日期：1998-11-01 00:00:00

abstract：:Evidence has documented the tumor-promoting properties of long non-coding RNA (lncRNA) FOXD2 adjacent opposite strand RNA 1 (FOXD2-AS1) in many cancers. However, little is known about its role in gallbladder cancer. Here, we aimed to characterize the functional relevance of lncRNA FOXD2-AS1 in gallbladder cancer and t...

journal_title：Gene therapy

authors： Gao J,Dai C,Yu X,Yin XB,Liao WJ,Huang Y,Zhou F

更新日期：2020-09-11 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：:Silencing of gene expression by small interfering RNAs (siRNAs) is rapidly becoming a powerful tool for genetic analysis of mammalian cells. The use of DNA-based plasmid vectors to achieve transient and stable expression of siRNA has been developed to avoid the problems of double-stranded oligonucleotides transfection...

journal_title：Gene therapy

authors： Sabbioni S,Callegari E,Manservigi M,Argnani R,Corallini A,Negrini M,Manservigi R

更新日期：2007-03-01 00:00:00

abstract：:Transplantation of allogeneic cells as well as of genetically corrected autologous cells are potent approaches to restore cellular functions in patients suffering from genetic diseases. The recipient's immune responses against non-self-antigens may compromise the survival of the grafted cells. Recipients of the graft ...

journal_title：Gene therapy

authors： Bots STF,Hoeben RC

更新日期：2017-07-01 00:00:00

abstract：:Synthetic oligonucleotides and DNA fragments of less than 1 kilobase (kb) have been shown to cause site-specific genetic alterations in mammalian cells in culture and in vivo. We have used a lacZ reporter gene system to compare the efficiency of episomal and chromosomal gene repair in human embryonic kidney epithelial...

journal_title：Gene therapy

authors： Nickerson HD,Colledge WH

更新日期：2003-09-01 00:00:00

abstract：:Advances in genetic analysis and a greater understanding of human immunodeficiency virus (HIV) molecular pathogenesis have identified critical viral targets for gene interference strategies. RNase P molecules have been proposed as a novel approach for gene targeting based upon their potent catalytic activity, as well ...

journal_title：Gene therapy

authors： Hnatyszyn H,Spruill G,Young A,Seivright R,Kraus G

更新日期：2001-12-01 00:00:00