Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library.

abstract：:We have tested the cationic liposome N-(1-(2,3-dioleoyloxy)propyl)-N,N,N-trimethyl-ammoniummethylsul phate, (DOTAP), for gene delivery in vitro and in vivo with a view to clinical use in gene therapy for cystic fibrosis. Delivery of lacZ cDNA-DOTAP complexes via aerosol showed promoter-dependent differences in the pat...

journal_title：Gene therapy

authors： McLachlan G,Davidson DJ,Stevenson BJ,Dickinson P,Davidson-Smith H,Dorin JR,Porteous DJ

更新日期：1995-11-01 00:00:00

abstract：:Cell surface targeting of recombinant adeno-associated virus (rAAV) vectors is an attractive strategy to modify AAV's natural tropism. As modification of the capsid surface is likely to affect the mechanism of vector internalization and consequently the vector's intracellular fate, we investigated early steps in cell ...

journal_title：Gene therapy

authors： Uhrig S,Coutelle O,Wiehe T,Perabo L,Hallek M,Büning H

更新日期：2012-02-01 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：:Antibodies have long been used in biomedical science as in vitro tools for the identification, purification and functional manipulation of target antigens; they have been exploited in vivo for diagnostic and therapeutic applications as well. Recent advances in antibody engineering have now allowed the genes encoding a...

journal_title：Gene therapy

authors： Marasco WA

更新日期：1997-01-01 00:00:00

abstract：:Based on observations that DBA/2 mice develop a highly specific response towards an HLA-Cw3-derived epitope, consisting entirely of CD8+CD62L-Vbeta10+ cells, we have established an in vivo mouse model for screening a variety of immunization approaches. Responder cells were readily detectable in small samples of the pe...

journal_title：Gene therapy

authors： Paster W,Zehetner M,Kalat M,Schüller S,Schweighoffer T

更新日期：2003-05-01 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 clinical efficacy and safety as well as the application range of gene therapy will be broadened by developing systems capable of finely modulating the expression of therapeutic genes. Transgene regulation will be crucial for maintaining appropriate levels of a gene product within the therapeutic range, thus preven...

journal_title：Gene therapy

authors： Toniatti C,Bujard H,Cortese R,Ciliberto G

更新日期：2004-04-01 00:00:00

abstract：:Lentivirus can be engineered to be a highly potent vector for gene therapy applications. However, generation of clinical grade vectors in enough quantities for therapeutic use is still troublesome and limits the preclinical and clinical experiments. As a first step to solve this unmet need we recently introduced a bac...

journal_title：Gene therapy

authors： Lesch HP,Laitinen A,Peixoto C,Vicente T,Makkonen KE,Laitinen L,Pikkarainen JT,Samaranayake H,Alves PM,Carrondo MJ,Ylä-Herttuala S,Airenne KJ

更新日期：2011-06-01 00:00:00

abstract：:This study uses a novel approach to gene therapy in which plasmid DNA is targeted to the pancreas in vivo using ultrasound-targeted microbubble destruction (UTMD) to achieve islet regeneration. Intravenous microbubbles carrying plasmids are destroyed within the pancreatic microcirculation by ultrasound, achieving loca...

journal_title：Gene therapy

authors： Chen S,Shimoda M,Wang MY,Ding J,Noguchi H,Matsumoto S,Grayburn PA

更新日期：2010-11-01 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：:The role of gap junctional intercellular communication (GJIC) in bystander killing with herpes simplex virus thymidine kinase (HSV-TK) and ganciclovir (GCV) was evaluated in U251 cells expressing a dominant-negative connexin 43 cDNA (DN14), and in HeLa cells, reportedly devoid of connexin protein. These cell lines bot...

journal_title：Gene therapy

authors： Gentry BG,Im M,Boucher PD,Ruch RJ,Shewach DS

更新日期：2005-07-01 00:00:00

abstract：:Several groups are assessing the use of cationic lipids for respiratory gene therapy. To date no human data are available regarding the safety of intra-pulmonary cationic lipid delivery. In preparation for a trial of pulmonary delivery of the CFTR gene, we have assessed the safety of nebulised lipid GL-67/DOPE/DMPE-PE...

journal_title：Gene therapy

authors： Chadwick SL,Kingston HD,Stern M,Cook RM,O'Connor BJ,Lukasson M,Balfour RP,Rosenberg M,Cheng SH,Smith AE,Meeker DP,Geddes DM,Alton EW

更新日期：1997-09-01 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：:Transplantation faces several major obstacles that could be overcome by expression of immunomodulatory proteins through application of gene therapy techniques. Gene therapy strategies to prolong graft survival involve gene transfer of immunosuppressive or graft-protecting molecules. Very promising results have been ob...

journal_title：Gene therapy

authors： Guillot C,Le Mauff B,Cuturi MC,Anegon I

更新日期：2000-01-01 00:00:00

abstract：:First-generation adenoviral (Ad) vectors are frequently used vectors for experimental and clinical gene transfer. Earlier it has been shown that parallel overexpression of the cell cycle regulator p21(Waf1/Cip1) (p21) or antiapoptotic bcl-2 from a second vector reduces cytotoxicity and improves transgene expression. H...

journal_title：Gene therapy

authors： Schumacher A,Horvat S,Woischwill C,Wolff G,Witt C

更新日期：2009-04-01 00:00:00

abstract：:Adoptive immunotherapy of cancer using chimeric antigen receptor (CAR)-engineered T cells with redirected specificity showed efficacy in recent trials. In preclinical models, 'second-generation' CARs with CD28 costimulatory domain in addition to CD3ζ performed superior in redirecting T-cell effector functions and surv...

journal_title：Gene therapy

authors： Chmielewski M,Hombach AA,Abken H

更新日期：2011-01-01 00:00:00

abstract：:Until recently, adeno-associated virus 9 (AAV9) was considered the AAV serotype most effective in crossing the blood-brain barrier (BBB) and transducing cells of the central nervous system (CNS), following systemic injection. However, a newly engineered capsid, AAV-PHP.B, is reported to cross the BBB at even higher ef...

journal_title：Gene therapy

authors： Rincon MY,de Vin F,Duqué SI,Fripont S,Castaldo SA,Bouhuijzen-Wenger J,Holt MG

更新日期：2018-04-01 00:00:00

abstract：:A retroviral vector constructed from the murine leukemia virus (MLV) can only express transgenes in cells undergoing mitosis, indicating its suitability as a delivery vehicle for cancer gene therapy. However, the transduction efficiency (TE) of retroviruses embedding endogenous envelope proteins in human cancer cells ...

journal_title：Gene therapy

authors： Lee H,Song JJ,Kim E,Yun CO,Choi J,Lee B,Kim J,Chang JW,Kim JH

更新日期：2001-02-01 00:00:00

abstract：:The structure of 'stabilized plasmid-lipid particles' (SPLP) and their properties as systemic gene therapy vectors has been investigated. We show that SPLP can be visualized employing cryo-electron microscopy to be homogeneous particles of diameter 72 +/- 5 nm consisting of a lipid bilayer surrounding a core of plasmi...

journal_title：Gene therapy

authors： Tam P,Monck M,Lee D,Ludkovski O,Leng EC,Clow K,Stark H,Scherrer P,Graham RW,Cullis PR

更新日期：2000-11-01 00:00:00

abstract：:The potential for gene therapy to be an effective treatment for cystic fibrosis has been hampered by the limited gene transfer efficiency of current vectors. We have shown that recombinant Sendai virus (SeV) is highly efficient in mediating gene transfer to differentiated airway epithelial cells, because of its capaci...

journal_title：Gene therapy

authors： Ferrari S,Griesenbach U,Iida A,Farley R,Wright AM,Zhu J,Munkonge FM,Smith SN,You J,Ban H,Inoue M,Chan M,Singh C,Verdon B,Argent BE,Wainwright B,Jeffery PK,Geddes DM,Porteous DJ,Hyde SC,Gray MA,Hasegawa M,Alton

更新日期：2007-10-01 00:00:00

abstract：:The annual rate of kidney graft loss caused by chronic allograft nephropathy (CAN) has not improved over the past decade. Recent reports suggest that acute renal ischemia results in development of CAN. The goal of the present study was to assess the renoprotective potential and safety of hepatocyte growth factor (HGF)...

journal_title：Gene therapy

authors： Isaka Y,Yamada K,Takabatake Y,Mizui M,Miura-Tsujie M,Ichimaru N,Yazawa K,Utsugi R,Okuyama A,Hori M,Imai E,Takahara S

更新日期：2005-05-01 00:00:00

abstract：:In-stent restenosis results exclusively from neointimal hyperplasia due to mechanical injury and a foreign body response to the prosthesis. Inflammation mediated by monocyte chemoattractant protein-1 (MCP-1) might therefore underlie in-stent restenosis. We recently devised a new strategy for anti-MCP-1 gene therapy by...

journal_title：Gene therapy

authors： Ohtani K,Usui M,Nakano K,Kohjimoto Y,Kitajima S,Hirouchi Y,Li XH,Kitamoto S,Takeshita A,Egashira K

更新日期：2004-08-01 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：: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：: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：:Current generation adenovirus (Ad) vectors are deleted for the E1 region of genes and require propagation in E1 expressing 293 cells. Expression of genes delivered by Ad vectors into immunocompetent hosts is generally transient since the current vectors are not completely replication defective. Viral proteins expresse...

journal_title：Gene therapy

authors： Amalfitano A,Chamberlain JS

更新日期：1997-03-01 00:00:00

abstract：:The development of lentiviral vectors that use Rev-independent mechanisms of nuclear export for their genomic RNA could facilitate the construction of novel anti-HIV vectors. We have improved the titers of Rev-independent lentiviral vectors having the SRV-1 CTE by mutating the major splice donor and acceptor sites pre...

journal_title：Gene therapy

authors： Mautino MR,Keiser N,Morgan RA

更新日期：2000-08-01 00:00:00

abstract：:Chronic infection with hepatitis B virus (HBV) puts individuals at high risk for complicating cirrhosis and liver cancer, but available treatment to counter the virus rarely eliminates infection. Although harnessing RNA interference (RNAi) to silence HBV genes has shown the potential, achieving efficient and durable s...

journal_title：Gene therapy

authors： Ivacik D,Ely A,Ferry N,Arbuthnot P

更新日期：2015-02-01 00:00:00

abstract：:Radiosensitization of human gastrointestinal tumors by 5-fluorouracil (5-FU) has been studied in vitro and clinically in human cancer therapy trials. The bacterial enzyme cytosine deaminase (CD) converts the nontoxic prodrug 5-fluorocytosine (5-FC) into 5-FU. Human colon cancer cells stably expressing CD have been sho...

journal_title：Gene therapy

authors： Stackhouse MA,Pederson LC,Grizzle WE,Curiel DT,Gebert J,Haack K,Vickers SM,Mayo MS,Buchsbaum DJ

更新日期：2000-06-01 00:00:00

abstract：:For the development of human immunodeficiency virus type 1 (HIV-1) vaccines, traditional approaches inducing virus-neutralizing antibodies have so far failed. Thus the effort is now focused on elicitation of cellular immunity. We are currently testing in clinical trials in the United Kingdom and East Africa a T-cell v...

journal_title：Gene therapy

authors： Nkolola JP,Wee EG,Im EJ,Jewell CP,Chen N,Xu XN,McMichael AJ,Hanke T

更新日期：2004-07-01 00:00:00