Messenger RNA electroporation is highly efficient in mouse embryonic stem cells: successful FLPe- and Cre-mediated recombination.

abstract：:The possibility of achieving multiple systemic expression of human interferon-beta in mice upon repeated intravenous administration of cationic liposome-DNA complex (lipoplex) was investigated. Lipoplexes containing the pentammonio lipid pcTG90 were first optimized by selecting the most efficient ratio of pcTG90 to ph...

journal_title：Gene therapy

authors： Meyer O,Schughart K,Pavirani A,Kolbe HV

更新日期：2000-09-01 00:00:00

abstract：:Human adenovirus (AV) is a favored vector for delivery of therapeutic genes into certain target cells, such as skeletal muscle cells for gene therapy. Here we show that replication-defective (E1 + E3 deleted) human type 5 adenovirus (AV) recombinants containing a reporter gene insert (RSV-luciferase or RSV-Lux) can ve...

journal_title：Gene therapy

authors： Acsadi G,Jani A,Huard J,Blaschuk K,Massie B,Holland P,Lochmüller H,Karpati G

更新日期：1994-09-01 00:00:00

abstract：:In 1998, two distinct and exciting scientific fields emerged which have profoundly shaped the current direction of biomedical research. The discovery of RNA interference (RNAi) and the derivation of human embryonic stem (ES) cells have yielded exciting new possibilities for researchers and clinicians alike. While fund...

journal_title：Gene therapy

authors： Heidersbach A,Gaspar-Maia A,McManus MT,Ramalho-Santos M

更新日期：2006-03-01 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：:Gene targeting by single-stranded oligodeoxyribonucleotides (ssODNs) is a promising technique for introducing site-specific sequence alterations without affecting the genomic organization of the target locus. Here, we discuss the significant progress that has been made over the last 5 years in unraveling the mechanism...

journal_title：Gene therapy

authors： Aarts M,te Riele H

更新日期：2011-03-01 00:00:00

abstract：:This study examines the clinical relevance of tissue engineering integrating gene therapy and polymer science to bone regeneration. Bilateral maxillary defects (3 x 1.2 cm(2)) in 20 miniature swine were bridged with a bioresorbable internal splint. Constructs were created using ex vivo adenovirus bone morphogenetic pr...

journal_title：Gene therapy

authors： Chang SC,Chuang HL,Chen YR,Chen JK,Chung HY,Lu YL,Lin HY,Tai CL,Lou J

更新日期：2003-11-01 00:00:00

abstract：:Gene therapy with viral vectors has progressed to clinical trials. However, the localization of viral vector delivery to diseased target sites remains a challenge. We tested the hypothesis that an adenoviral vector could be successfully delivered by complexation with a specific antibody that is bound to a biodegradabl...

journal_title：Gene therapy

authors： Levy RJ,Song C,Tallapragada S,DeFelice S,Hinson JT,Vyavahare N,Connolly J,Ryan K,Li Q

更新日期：2001-05-01 00:00:00

abstract：:Production of retroviral vectors for clinical use requires removal of cells and cellular debris. We combined a series of filters of decreasing pore size using commercially available blood banking filters approved for clinical use. The collection bag and filters can be connected to create a sterile, closed system using...

journal_title：Gene therapy

authors： Reeves L,Cornetta K

更新日期：2000-12-01 00:00:00

abstract：:Gene therapy is thought to be a promising method for the treatment of various diseases. One gene therapy strategy involves the manipulations on a process of formation of new vessels, commonly defined as angiogenesis. Angiogenic and antiangiogenic gene therapy is a new therapeutic approach to the treatment of cardiovas...

journal_title：Gene therapy

authors： Malecki M,Kolsut P,Proczka R

更新日期：2005-10-01 00:00:00

abstract：:The sodium iodide symporter (NIS) mediates iodide uptake into thyrocytes and is the molecular basis of thyroid radioiodine therapy. We previously have shown that NIS gene transfer into the F98 rat gliomas facilitated tumor imaging and increased survival by radioiodine. In this study, we show that: (1) the therapeutic ...

journal_title：Gene therapy

authors： Shen DH,Marsee DK,Schaap J,Yang W,Cho JY,Hinkle G,Nagaraja HN,Kloos RT,Barth RF,Jhiang SM

更新日期：2004-01-01 00:00:00

abstract：:Intramuscular (i.m.) injection of a plasmid encoding human carcinoembryonic antigen (CEA) elicited immunity against transplanted syngeneic (C57BL/6) CEA-positive Lewis lung carcinoma (CEA/LLC) cells, but tumors still appeared in all mice. In wild-type mice, coinjection of an IL-12 plasmid markedly enhanced anti-CEA hu...

journal_title：Gene therapy

authors： Song K,Chang Y,Prud'homme GJ

更新日期：2000-09-01 00:00:00

abstract：:Mesenchymal stem cells (MSC) are a group of clonogenic cells present among the bone marrow stroma and capable of multilineage differentiation into mesoderm-type cells such as osteoblasts, adipocytes and chondrocytes. Due to their ease of isolation and their differentiation potential, MSC are being introduced into clin...

journal_title：Gene therapy

authors： Abdallah BM,Kassem M

更新日期：2008-01-01 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 report on systemic delivery and long-term biological effects of apolipoprotein E (apoE) obtained by intramuscular (i.m.) plasmid DNA injection. ApoE plays an important role in lipoprotein catabolism and apoE knock-out mice develop severe hypercholesterolemia and diffuse atherosclerosis. We have injected apoE-defici...

journal_title：Gene therapy

authors： Rinaldi M,Catapano AL,Parrella P,Ciafrè SA,Signori E,Seripa D,Uboldi P,Antonini R,Ricci G,Farace MG,Fazio VM

更新日期：2000-11-01 00:00:00

abstract：:The role of two receptors, previously proposed to mediate the entry of adenoviruses into human cells, the coxsackie and adenovirus receptor (CAR) and the major histocompatibility complex (MHC) class I heavy chain has been investigated. The expression of MHC class I in many tumours is reduced or absent, therefore if th...

journal_title：Gene therapy

authors： McDonald D,Stockwin L,Matzow T,Blair Zajdel ME,Blair GE

更新日期：1999-09-01 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：:This report examines the commercialization of gene therapy in the context of innovation theories that posit a relationship between the maturation of a technology through its life cycle and prospects for successful product development. We show that the field of gene therapy has matured steadily since the 1980s, with th...

journal_title：Gene therapy

authors： Ledley FD,McNamee LM,Uzdil V,Morgan IW

更新日期：2014-02-01 00:00:00

abstract：:Most neoplasms do not induce antitumor immune responses that can control tumor growth. Tumor associated antigens (TAAs) are insufficiently immunogenic. A vaccine that augments the immunogenic properties of TAAs could be of importance in the treatment of cancer patients. In an animal model, we prepared a vaccine by tra...

journal_title：Gene therapy

authors： de Zoeten EF,Markovic D,Cohen EP

更新日期：2002-09-01 00:00:00

abstract：:As soon as RNA interference (RNAi) was found to work in mammalian cells, research quickly focused on harnessing this powerful endogenous and specific mechanism of gene silencing for human therapy. RNAi uses small RNAs, less than 30 nucleotides in length, to suppress expression of genes with complementary sequences. Tw...

journal_title：Gene therapy

authors： Dykxhoorn DM,Palliser D,Lieberman J

更新日期：2006-03-01 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：:We tested the longevity of gene expression provided by autonomously replicating vectors. The vectors contain segments of human genomic DNA that provide efficient replication initiation and sequences derived from Epstein-Barr virus that provide nuclear retention. In order to monitor gene expression, the vectors also ca...

journal_title：Gene therapy

authors： Wohlgemuth JG,Kang SH,Bulboaca GH,Nawotka KA,Calos MP

更新日期：1996-06-01 00:00:00

abstract：:Isolated limb perfusion (ILP) is a limb salvage surgical modality used to deliver chemotherapy and biologic agents to locally advanced and recurrent extremity soft tissue sarcoma (STS), and may be readily tailored for delivery of gene therapy. We set out to test the feasibility of delivering AdFLAGp53 (replication inc...

journal_title：Gene therapy

authors： Hannay J,Davis JJ,Yu D,Liu J,Fang B,Pollock RE,Lev D

更新日期：2007-04-01 00:00:00

abstract：:Human adipose tissue mesenchymal stromal cells (AMSCs) share common traits, including similar differentiation potential and cell surface markers, with their bone marrow counterparts. Owing to their general availability, higher abundance and ease of isolation AMSCs may be convenient autologous delivery vehicles for loc...

journal_title：Gene therapy

authors： Vilalta M,Dégano IR,Bagó J,Aguilar E,Gambhir SS,Rubio N,Blanco J

更新日期：2009-04-01 00:00:00

abstract：:Mobilized blood CD34+ cells from cancer patients were ex vivo infected by a recombinant adenovirus vector carrying an alkaline phosphatase gene, whose expression is evaluable by flow cytometry. A mean of 40% CD34+ cells were infected by the vector, with high levels of expression of the transgene. Among attempts to imp...

journal_title：Gene therapy

authors： Bregni M,Shammah S,Malaffo F,Di Nicola M,Milanesi M,Magni M,Matteucci P,Ravagnani F,Jordan CT,Siena S,Gianni AM

更新日期：1998-04-01 00:00:00

abstract：:A replication-incompetent adenoviral vector encoding the heavy chain C-fragment (H(C)50) of botulinum neurotoxin type C (BoNT/C) was evaluated as a mucosal vaccine against botulism in a mouse model. Single intranasal inoculation of the adenoviral vector elicited a high level of H(C)50-specific IgG, IgG1 and IgG2a in s...

journal_title：Gene therapy

authors： Xu Q,Pichichero ME,Simpson LL,Elias M,Smith LA,Zeng M

更新日期：2009-03-01 00:00:00

abstract：:Advances in islet transplantation have encouraged efforts to create alternative insulin-secreting cells that overcome limitations associated with current therapies. We have recently demonstrated durable correction of murine and porcine diabetes by syngeneic and autologous implantation, respectively, of primary hepatoc...

journal_title：Gene therapy

authors： Chen NK,Tan SY,Udolph G,Kon OL

更新日期：2010-05-01 00:00:00

abstract：:Coagulation factor X (FX)-binding ablated adenovirus type 5 (Ad5) vectors have been genetically engineered to ablate the interaction with FX, resulting in substantially reduced hepatocyte transduction following intravenous administration in rodents. Here, we quantify viral genomes and gene transfer mediated by Ad5 and...

journal_title：Gene therapy

authors： Alba R,Bradshaw AC,Mestre-Francés N,Verdier JM,Henaff D,Baker AH

更新日期：2012-01-01 00:00:00

abstract：:Dendritic cells (DC) are among the most potent antigen-presenting cells known and play an important role in the initiation of antigen-specific T-lymphocyte responses. Several recent studies have demonstrated that DC expressing vector-encoded tumor-associated antigens can induce protective and therapeutic immunity in m...

journal_title：Gene therapy

authors： Diao J,Smythe JA,Smyth C,Rowe PB,Alexander IE

更新日期：1999-05-01 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：: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