Liver bypass significantly increases the transduction efficiency of recombinant adenoviral vectors in the lung, intestine, and kidney.

abstract：:Due to both the avascularity of the cornea and the relatively immune-privileged status of the eye, corneal transplantation is one of the most successful clinical transplant procedures. However, in high-risk patients, which account for >20% of the 180,000 transplants carried out worldwide each year, the rejection rate ...

journal_title：Human gene therapy

authors： Fouladi N,Parker M,Kennedy V,Binley K,McCloskey L,Loader J,Kelleher M,Mitrophanous KA,Stout JT,Ellis S

更新日期：2018-06-01 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 liver is an attractive target tissue for gene therapy. Current approaches for hepatic gene delivery include retroviral and adenoviral vectors, liposome/DNA, and peptide/DNA complexes. This study describes a technique for direct injection of DNA into liver that led to significant gene expression. Gene expression wa...

journal_title：Human gene therapy

authors： Hickman MA,Malone RW,Lehmann-Bruinsma K,Sih TR,Knoell D,Szoka FC,Walzem R,Carlson DM,Powell JS

更新日期：1994-12-01 00:00:00

abstract：:Production of clinical-grade gammaretroviral vectors for ex vivo gene delivery requires a scalable process that can rapidly generate large amounts of vector supernatant, clear large numbers of residual packaging cells with minimal decreases in vector titer, and satisfy all current regulatory guidelines regarding produ...

journal_title：Human gene therapy

authors： Feldman SA,Goff SL,Xu H,Black MA,Kochenderfer JN,Johnson LA,Yang JC,Wang Q,Parkhurst MR,Cross S,Morgan RA,Cornetta K,Rosenberg SA

更新日期：2011-01-01 00:00:00

abstract：:Despite improvements in drug and device therapy for heart failure, hospitalization rates and mortality have changed little in the past decade. Randomized clinical trials using gene transfer to improve function of the failing heart are the focus of this review. Four randomized clinical trials of gene transfer in heart ...

journal_title：Human gene therapy

authors： Penny WF,Hammond HK

更新日期：2017-05-01 00:00:00

abstract：:Adenoviruses (Ads) have shown great utility as vectors for the delivery of genes to mammalian cells, partly because of their ability to infect a wide range of different cell types independent of the replicative state of the cell. However, Ads do not transduce mature muscle efficiently because of low levels of the natu...

journal_title：Human gene therapy

authors： Bramson JL,Grinshtein N,Meulenbroek RA,Lunde J,Kottachchi D,Lorimer IA,Jasmin BJ,Parks RJ

更新日期：2004-02-01 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：:Nonviral jet injection is an applicable technology for in vivo gene transfer of naked DNA. However, little is known about the biodistribution and clearance of jet-injected DNA, or about its localization within tissue and cells. Therefore, in this study we analyzed the intratumoral and systemic biodistribution of jet-i...

journal_title：Human gene therapy

authors： Walther W,Minow T,Martin R,Fichtner I,Schlag PM,Stein U

更新日期：2006-06-01 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：:The recombinant adenoviral (Ad) vector is being considered as a cancer vaccine platform because it efficiently induces immune responses to tumor antigens by intradermal immunization. The aims of this study were to evaluate the potential toxicities and biodistribution after a single dose or six weekly intradermal doses...

journal_title：Human gene therapy

authors： Plog MS,Guyre CA,Roberts BL,Goldberg M,St George JA,Perricone MA

更新日期：2006-07-01 00:00:00

abstract：:Diabetes mellitus derives from either insulin deficiency (type I) or resistance (type II). Homozygous mutations in the insulin receptor (IR) gene cause the rare leprechaunism and Rabson-Mendenhall syndromes, severe forms of hyperinsulinemic insulin resistance for which no therapy is currently available. Systems have b...

journal_title：Human gene therapy

authors： Cotugno G,Pollock R,Formisano P,Linher K,Beguinot F,Auricchio A

更新日期：2004-11-01 00:00:00

abstract：:Hereditary tyrosinemia type 1 (HT1) is an autosomal recessive disorder caused by deficiency of fumarylacetoacetate hydrolase (FAH). It has been previously shown that ex vivo hepatocyte-directed gene therapy using an integrating lentiviral vector to replace the defective Fah gene can cure liver disease in small- and la...

journal_title：Human gene therapy

authors： VanLith C,Guthman R,Nicolas CT,Allen K,Du Z,Joo DJ,Nyberg SL,Lillegard JB,Hickey RD

更新日期：2018-11-01 00:00:00

abstract：:Adenovirus-polylysine-DNA complexes were evaluated for their capacity to accomplish direct in vivo gene transfer to airway epithelium employing a rodent model. Binary complexes containing transferrin or adenovirus, or combination complexes containing both transferrin and adenovirus, were evaluated. The highest in vitr...

journal_title：Human gene therapy

authors： Gao L,Wagner E,Cotten M,Agarwal S,Harris C,Rømer M,Miller L,Hu PC,Curiel D

更新日期：1993-02-01 00:00:00

abstract：:Mice bearing breast tumors were treated with a single dose of an adenovirus expressing interleukin-12 (AdmIL-12.1) injected intratumorally, which produced regressions in greater than 75% of the treated tumors; approximately one-third of the animals remained tumor free. Complete regression was associated with immunity ...

journal_title：Human gene therapy

authors： Bramson JL,Hitt M,Addison CL,Muller WJ,Gauldie J,Graham FL

更新日期：1996-10-20 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：:Deficiencies in different steps of purine metabolism give rise to a number of human inherited disorders. Lesch-Nyhan syndrome is a severe neurological disorder, caused by a deficiency in the purine salvage enzyme hypoxanthine phosphoribosyltransferase (HPRT). HPRT-deficient mice have been generated, but have proved to...

journal_title：Human gene therapy

authors： Redhead NJ,Selfridge J,Wu CL,Melton DW

更新日期：1996-08-20 00:00:00

abstract：:Advances in cell and gene therapy are opening up new avenues for regenerative medicine. Because of their acquired pluripotency, human induced pluripotent stem cells (hiPSCs) are a promising source of autologous cells for regenerative medicine. They show unlimited self-renewal while retaining the ability, in principle,...

journal_title：Human gene therapy

authors： Garate Z,Davis BR,Quintana-Bustamante O,Segovia JC

更新日期：2013-06-01 00:00:00

abstract：:Glycogen storage disease type II (GSDII) is a lysosomal storage disease caused by a deficiency in acid alpha-glucosidase (GAA), and leads to cardiorespiratory failure by the age of 2 years. In this study, we investigate the impact of anti-GAA antibody formation on cross-correction of the heart, diaphragm, and hind-lim...

journal_title：Human gene therapy

authors： Cresawn KO,Fraites TJ,Wasserfall C,Atkinson M,Lewis M,Porvasnik S,Liu C,Mah C,Byrne BJ

更新日期：2005-01-01 00:00:00

abstract：:Combining chemotherapy and immunotherapy is problematic because chemotherapy can ablate the immune responses initiated by modulators of the immune system. We hypothesized that protection of immunocompetent cells from the toxic effects of chemotherapy, using drug resistance gene therapy strategies, would allow the comb...

journal_title：Human gene therapy

authors： McMillin DW,Hewes B,Gangadharan B,Archer DR,Mittler RS,Spencer HT

更新日期：2006-08-01 00:00:00

abstract：:In the next decades, gene editing technologies are expected to be used in the treatment and prevention of human diseases. Yet, the future uses of gene editing in medicine are still unknown, including its applicability and effectiveness to the treatment and prevention of infectious diseases, cancer, and monogenic and p...

journal_title：Human gene therapy

authors： Rocha LFM,Braga LAM,Mota FB

更新日期：2020-08-01 00:00:00

abstract：:Targeted vectors provide a number of advantages for systemic and local gene delivery strategies. Several groups have investigated the utility of using various ligands to alter the tropism of adenovirus (Ad) vectors. We have previously demonstrated that fibroblast growth factor (FGF) ligands can specifically target DNA...

journal_title：Human gene therapy

authors： Printz MA,Gonzalez AM,Cunningham M,Gu DL,Ong M,Pierce GF,Aukerman SL

更新日期：2000-01-01 00:00:00

abstract：:Activation of T cells is necessary for efficient retroviral-mediated gene transfer. In addition, if the population of infused cells is to be limited to transduced cells, a means of positive selection is required. We describe a clinical scale procedure for activation of donor T cells with anti-CD3/CD28 beads followed b...

journal_title：Human gene therapy

authors： Orchard PJ,Blazar BR,Burger S,Levine B,Basso L,Nelson DM,Gordon K,McIvor RS,Wagner JE,Miller JS

更新日期：2002-05-20 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：:The objective of this phase II investigation is to assess the safety and efficacy of a plasmid mediated approach to induce angiogenesis/arteriogenesis with the angiomatrix protein Del-1 (developmentally regulated endothelial locus 1), in subjects with intermittent claudication (IC) secondary to peripheral arterial dis...

journal_title：Human gene therapy

authors： Rajagopalan S,Olin JW,Young S,Erikson M,Grossman PM,Mendelsohn FO,Regensteiner JG,Hiatt WR,Annex BH

更新日期：2004-06-01 00:00:00

abstract：:Adeno-associated virus (AAV) gene transfer is a promising treatment for genetic abnormalities. Optimal AAV vectors are showing success in clinical trials. Gene transfer to skeletal muscle and liver is being explored as a potential therapy for some conditions, that is, α1-antitrypsin (AAT) disorder and hemophilia B. Ex...

journal_title：Human gene therapy

authors： Carrig S,Bijjiga E,Wopat MJ,Martino AT

更新日期：2016-11-01 00:00:00

abstract：:One of the major obstacles to pulmonary-directed gene therapy using adenoviral vectors is the induction of inflammation. We investigated whether the adenoviral particles that constitute the initial inoculum can serve as an inflammatory stimulus, independent of their ability to express genes that they contain. Viral pa...

journal_title：Human gene therapy

authors： McCoy RD,Davidson BL,Roessler BJ,Huffnagle GB,Janich SL,Laing TJ,Simon RH

更新日期：1995-12-01 00:00:00

abstract：:Airway infiltration by eosinophils is a major characteristic of chronic asthma. CCL11 (eotaxin-1) is secreted by lung epithelial cells and functions as the major chemokine for eosinophil recruitment. Pseudotyped adeno-associated virus (AAV) 2/9, composed by the AAV2 rep and AAV9 cap genes, can efficiently target lung ...

journal_title：Human gene therapy

authors： Wu CJ,Huang WC,Chen LC,Shen CR,Kuo ML

更新日期：2012-11-01 00:00:00

abstract：:The combination of immunization strategies with gene therapy methods constitutes a powerful tool for the purpose of genetic immunization. The cutaneous microenvironment, rich in professional antigen-presenting cells and accessory cells capable of initiating and controlling the intensity of specific immune responses, m...

journal_title：Human gene therapy

authors： Larregina AT,Falo LD Jr

更新日期：2000-11-01 00:00:00

abstract：:The transfer of a drug resistance gene into hematopoietic cells is an approach being investigated to overcome the problem of myelosuppression produced by anticancer drugs. Chemotherapeutic agents are often given in combination in order to increase their effectiveness. Consequently, there is an advantage in designing v...

journal_title：Human gene therapy

authors： Beauséjour CM,Le NL,Létourneau S,Cournoyer D,Momparler RL

更新日期：1998-11-20 00:00:00

abstract：:Hematopoietic stem cells (HSCs) are a potential target for the retrovirus-mediated transfer of chemotherapeutic drug resistance genes. For integration of the proviral DNA in the HSC genome cell division is required. In the bone marrow (BM) hematopoiesis occurs in the vicinity of stroma cells. Soluble stroma components...

journal_title：Human gene therapy

authors： Schiedlmeier B,Buss EC,Veldwijk MR,Zeller WJ,Fruehauf S

更新日期：1999-06-10 00:00:00