Preexisting antiadenoviral immunity is not a barrier to efficient and stable transduction of the brain, mediated by novel high-capacity adenovirus vectors.

abstract：:Oncolytic viruses (OV) are novel cancer gene therapies that are moving toward the forefront of modern medicines. However, their full therapeutic potential is hindered by the lack of convenient and reliable strategies to visualize and quantify OV growth kinetics and therapeutic efficacy in live cells. Here, we present ...

journal_title：Human gene therapy

authors： Quillien L,Top S,Kappler S,Redouté A,Dusetti N,Quentin-Froignant C,Lulka H,Camus C,Buscail L,Gallardo F,Bertagnoli S,Cordelier P

更新日期：2021-01-27 00:00:00

abstract：:Targeted therapy produces objective responses in bladder cancer patients, although the responses can be short. Meanwhile, response rates to immune therapy are lower, but the effects are more durable. Based on these findings, it was hypothesized that urothelial carcinoma associated 1 (UCA1)-targeted therapy could syner...

journal_title：Human gene therapy

authors： Zhen S,Lu J,Chen W,Zhao L,Li X

更新日期：2018-12-01 00:00:00

abstract：:Protein transduction domains (PTD), which can transport proteins or peptides across biological membranes, have been identified in several proteins of viral, invertebrate, and vertebrate origin. Here, we evaluate the immunological and biological consequences of including PTD in synthetic peptides and in DNA vaccines th...

journal_title：Human gene therapy

authors： Leifert JA,Lindencrona JA,Charo J,Whitton JL

更新日期：2001-10-10 00:00:00

abstract：:Low in vivo transduction efficiency and safety concerns have been hurdles for effective hematopoietic stem cell (HSC) gene therapy. Here, we investigate whether the safety and efficiency of retroviral gene transfer into HSCs can be improved by using human allogeneic umbilical cord blood (UCB)-derived supplements inste...

journal_title：Human gene therapy

authors： Moon N,Yang SJ,Park BB,Chung YS,Lee JW,Oh IH

更新日期：2008-07-01 00:00:00

abstract：:Lentiviral vectors are promising tools for gene transfer into the central nervous system. We have characterized in detail transduction with human immunodeficiency virus type 1 (HIV-1)-derived vectors encoding enhanced green fluorescent protein (eGFP) in the adult mouse brain. Different brain regions such as the striat...

journal_title：Human gene therapy

authors： Baekelandt V,Claeys A,Eggermont K,Lauwers E,De Strooper B,Nuttin B,Debyser Z

更新日期：2002-05-01 00:00:00

abstract：:pZIG(hGCSFR) is a retroviral vector that can co-express two genes and also provides alternative selection markers. This retroviral vector has been constructed to incorporate an internal ribosome entry site (IRES) element to co-express two exogenous genes in mammalian cells. Two marker/selection genes have been cloned ...

journal_title：Human gene therapy

authors： Saleh M

更新日期：1997-05-20 00:00:00

abstract：:Abstract Our studies have shown that coinjection of conventional single-stranded adeno-associated virus 2 (ssAAV2) vectors carrying the enhanced green fluorescent protein (EGFP) gene with self-complementary (sc) AAV2-T cell protein tyrosine phosphatase (TC-PTP) and scAAV2-protein phosphatase-5 (PP5) vectors resulted i...

journal_title：Human gene therapy

authors： Jayandharan GR,Zhong L,Sack BK,Rivers AE,Li M,Li B,Herzog RW,Srivastava A

更新日期：2010-03-01 00:00:00

abstract：:NKG2D ligands (NKG2DLs) are widely expressed on ovarian cancers to various degrees, making them attractive targets for immunotherapy. Here, we applied a chimeric antigen receptor (CAR) approach for the targeting of NKG2DLs expressed on human ovarian cancer cells and evaluated the impact of pharmacological upregulation...

journal_title：Human gene therapy

authors： Song DG,Ye Q,Santoro S,Fang C,Best A,Powell DJ Jr

更新日期：2013-03-01 00:00:00

abstract：:This is an erratum of the published paper "Preclinical Evaluation of Chimeric Antigen Receptor-Modified T Cells Specific to Epithelial Cell Adhesion Molecule for Treating Colorectal Cancer". There are some errors in figure 6C and 7C in the article due to authors' mistakes when preparing the figures. Specifically, repr...

journal_title：Human gene therapy

authors： Wang W

更新日期：2019-08-01 00:00:00

abstract：:Recombinant adenoviruses have received much attention as a potential vector for gene therapy because of their ability to transduce many cell types with high efficiencies in vivo. After intravenous infusion, the majority of the vector is found in hepatocytes, but vector DNA is found to varying degrees in other tissues....

journal_title：Human gene therapy

authors： Peeters MJ,Patijn GA,Lieber A,Meuse L,Kay MA

更新日期：1996-09-10 00:00:00

abstract：:Malignant pleural mesothelioma (MPM) is a fatal disease with a median survival of less than 14 months. For the first time, a genetically engineered vaccinia virus is shown to produce efficient infection, replication, and oncolytic effect against MPM. GLV-1h68 is a replication-competent engineered vaccinia virus carryi...

journal_title：Human gene therapy

authors： Kelly KJ,Woo Y,Brader P,Yu Z,Riedl C,Lin SF,Chen N,Yu YA,Rusch VW,Szalay AA,Fong Y

更新日期：2008-08-01 00:00:00

abstract：:Replication-competent adenoviruses may provide a highly efficient means of delivering therapeutic genes to tumors. Previously, we evaluated in vitro a replication-competent adenovirus (Ad5-CD/TKrep) containing a cytosine deaminase (CD)/herpes simplex type 1 thymidine kinase (HSV-1 TK) fusion gene that allows lytic vir...

journal_title：Human gene therapy

authors： Rogulski KR,Wing MS,Paielli DL,Gilbert JD,Kim JH,Freytag SO

更新日期：2000-01-01 00:00:00

abstract：:Clinical gene transfer research has involved adult and child subjects, and it is expected that gene transfer in fetal subjects will occur in the future. Some genetic diseases have serious adverse effects on the fetus before birth, and there is hope that prenatal gene therapy could prevent such disease progression. Res...

journal_title：Human gene therapy

authors： Strong C

更新日期：2011-11-01 00:00:00

abstract：:The ability to control proliferation of grafted cells in the heart and consequent graft size could dramatically improve the efficacy of cell therapies for cardiac repair. To achieve targeted graft cell proliferation, we created a chimeric receptor (F36Vfgfr-1) composed of a modified FK506-binding protein (F36V) fused ...

journal_title：Human gene therapy

authors： Stevens KR,Rolle MW,Minami E,Ueno S,Nourse MB,Virag JI,Reinecke H,Murry CE

更新日期：2007-05-01 00:00:00

abstract：:At present there is no known effective pharmacological therapy for acute lung injury (ALI). Because keratinocyte growth factor (KGF) promotes epithelial cell growth, intratracheal administration of KGF has the possibility of restoring lung tissue integrity in injured lungs and improving patient outcomes. However, trea...

journal_title：Human gene therapy

authors： Baba Y,Yazawa T,Kanegae Y,Sakamoto S,Saito I,Morimura N,Goto T,Yamada Y,Kurahashi K

更新日期：2007-02-01 00:00:00

abstract：:Retrovirus integration into the host cell genome occurs most efficiently in replicating cells. In agreement with this notion, it was observed that the efficiency with which hemopoietic stem cells (HSC) can be transduced is greatly enhanced when the hemopoietic growth factor (HGF) interleukin 3 (IL-3) is added to co-cu...

journal_title：Human gene therapy

authors： Einerhand MP,Bakx TA,Valerio D

更新日期：1991-01-01 00:00:00

abstract：:Human papillomavirus type 16 (HPV16) is associated with the development of anogenital cancers and their precursor lesions, intraepithelial neoplasia. Treatment strategies against HPV-induced intraepithelial neoplasia are not HPV specific and mostly consist of physical removal or ablation of lesions. We had previously ...

journal_title：Human gene therapy

authors： Sharma R,Palefsky JM

更新日期：2010-07-01 00:00:00

abstract：:Kallistatin is a serine proteinase inhibitor that has been shown to reduce joint swelling and to inhibit inflammation in a rat model of arthritis. In this study, we investigated the effect and mechanisms of kallistatin on cardiac function after myocardial ischemia-reperfusion (I/R) injury. The human kallistatin gene i...

journal_title：Human gene therapy

authors： Chao J,Yin H,Yao YY,Shen B,Smith RS Jr,Chao L

更新日期：2006-12-01 00:00:00

abstract：:Establishing pharmacological parameters, such as efficacy, routes of administration, and toxicity, for recombinant adeno-associated virus (rAAV) vectors is a prerequisite for gaining acceptance for clinical applications. In fact, even a therapeutic window, that is, the dose range between therapeutic efficacy and toxic...

journal_title：Human gene therapy

authors： Virag T,Cecchini S,Kotin RM

更新日期：2009-08-01 00:00:00

abstract：:Dystrophic epidermolysis bullosa (DEB) comprises a family of inherited blistering skin disorders for which no corrective therapy currently exists. In the most severe form, the Hallopeau-Siemens subtype (RDEB-HS), the epidermal adhesion protein collagen VII is absent from the skin as a consequence of null mutations in ...

journal_title：Human gene therapy

authors： Mecklenbeck S,Compton SH,Mejía JE,Cervini R,Hovnanian A,Bruckner-Tuderman L,Barrandon Y

更新日期：2002-09-01 00:00:00

abstract：:Isolated methylmalonic acidemia (MMA), a group of autosomal recessive inborn errors of metabolism, is most commonly caused by complete (mut(0)) or partial (mut(-)) deficiency of the enzyme methylmalonyl-CoA mutase (MUT). The severe metabolic instability and increased mortality experienced by many affected individuals,...

journal_title：Human gene therapy

authors： Harrington EA,Sloan JL,Manoli I,Chandler RJ,Schneider M,McGuire PJ,Calcedo R,Wilson JM,Venditti CP

更新日期：2016-05-01 00:00:00

abstract：:Three-vessel disease (TVD) is a severe coronary heart disease (CHD) with poor prognosis. Niemann-Pick C1-like 1 (NPC1L1) is a transporter protein for exogenous cholesterol absorption, and 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) is a rate-limiting enzyme for cholesterol synthesis. We aimed to investigat...

journal_title：Human gene therapy

authors： Zhao X,Li J,Tang X,Liu R,Xu J,Xu L,Jiang L,Huang K,Tian J,Feng X,Wu Y,Zhang Y,Wang D,Sun K,Xu B,Zhao W,Hui R,Gao R,Song L,Yuan J

更新日期：2021-01-22 00:00:00

abstract：:Skeletal muscle represents an attractive target tissue for adenoviral gene therapy to treat muscle disorders and as a production platform for systemic expression of therapeutic proteins. However, adenovirus serotype 5 vectors do not efficiently transduce adult muscle tissue. Here we evaluated whether capsid modificati...

journal_title：Human gene therapy

authors： Guse K,Suzuki M,Sule G,Bertin TK,Tyynismaa H,Ahola-Erkkilä S,Palmer D,Suomalainen A,Ng P,Cerullo V,Hemminki A,Lee B

更新日期：2012-10-01 00:00:00

abstract：:The purpose of this study was to assess the therapeutic potential of injecting the gene for HLA-B7/beta2-microglobulin into the subcutaneous metastatic nodules of patients who are refractory to conventional treatments. The nine patients evaluated were divided into three groups and given escalating doses of DNA (20, 40...

journal_title：Human gene therapy

authors： Heo DS,Yoon SJ,Kim WS,Lee KH,Seol JG,Lee SG,Jung CW,Cho EK,Kim CW,Park MH,Sung MW,Kim KH,Bang YJ,Kim NK

更新日期：1998-09-20 00:00:00

abstract：:With the aim of developing new gene transfer tools for treating CF with gene therapy, we have synthesized a novel family of molecules named cationic phosphonolipids. The most efficient among them were selected by in vitro screening to compare their activities in vivo in mouse lungs. We used a reporter gene whose activ...

journal_title：Human gene therapy

authors： Guillaume-Gable C,Floch V,Mercier B,Audrézet MP,Gobin E,Le Bolch G,Yaouanc JJ,Clément JC,des Abbayes H,Leroy JP,Morin V,Férec C

更新日期：1998-11-01 00:00:00

abstract：:The enzyme hypoxanthine-guanine phosphoribosyltransferase (HGPRT) expressed by the parasite Trypanosoma brucei (Tb) can convert allopurinol, a purine analogue, to corresponding nucleotides with greater efficiency than its human homologue. We have developed a retroviral system that expresses the parasitic enzyme and te...

journal_title：Human gene therapy

authors： Trudeau C,Yuan S,Galipeau J,Benlimame N,Alaoui-Jamali MA,Batist G

更新日期：2001-09-01 00:00:00

abstract：:Restoration of correct splicing of βIVS2-654-globin pre-mRNA was previously accomplished in erythroid cells from β-thalassemia/HbE patients by an engineered U7 small nuclear RNA (snRNA) that carried a sequence targeted to the cryptic branch point and an exonic splicing enhancer, U7.BP+623 snRNA. In this study, this ap...

journal_title：Human gene therapy

authors： d'Arqom A,Nualkaew T,Jearawiriyapaisarn N,Kole R,Svasti S

更新日期：2020-11-02 00:00:00

abstract：:The use of synthetic gene delivery systems in human gene transfer is hampered by poor transfection efficiencies, largely because of the inability of DNA to translocate across the nuclear pore complex. A means to overcome this barrier is to bind the DNA to nuclear localization signals (NLSs), which are recognized by sh...

journal_title：Human gene therapy

authors： Mesika A,Kiss V,Brumfeld V,Ghosh G,Reich Z

更新日期：2005-02-01 00:00:00

abstract：:Laminin-5 is composed of three distinct polypeptides, alpha3, beta3, and gamma2, which are encoded by three different genes, LAMA3, LAMB3, and LAMC2, respectively. We have isolated epidermal keratinocytes from a patient presenting with a lethal form of junctional epidermolysis bullosa characterized by a homozygous mut...

journal_title：Human gene therapy

authors： Dellambra E,Vailly J,Pellegrini G,Bondanza S,Golisano O,Macchia C,Zambruno G,Meneguzzi G,De Luca M

更新日期：1998-06-10 00:00:00

abstract：:An RNA melanoma vaccine was investigated to induce protective immunity in a mouse-melanoma model. LacZ mRNA was synthesized in vitro by pSFV3 expression vector and introduced into the spleen of mice, using HVJ-liposomes. A high level of beta-galactosidase activity was detected for 10 days in mouse spleen. The human me...

journal_title：Human gene therapy

authors： Zhou WZ,Hoon DS,Huang SK,Fujii S,Hashimoto K,Morishita R,Kaneda Y

更新日期：1999-11-01 00:00:00