Towards hematopoietic stem cell-mediated protection against infection with human immunodeficiency virus.

abstract：:Intracerebral administration of recombinant adeno-associated vector (AAV) has been performed in several clinical trials. However, delivery into the brain requires multiple injections and is not efficient to target the spinal cord, thus limiting its applications. To assess widespread and less invasive strategies, we te...

journal_title：Gene therapy

authors： Hordeaux J,Dubreil L,Deniaud J,Iacobelli F,Moreau S,Ledevin M,Le Guiner C,Blouin V,Le Duff J,Mendes-Madeira A,Rolling F,Cherel Y,Moullier P,Colle MA

更新日期：2015-04-01 00:00:00

abstract：UNLABELLED:The aim of this study was to determine the effect of RNA interference inhibition of mineralocorticoid receptor (MR) on cold-induced hypertension (CIH) and renal damage. Recombinant adeno-associated virus (AAV) carrying short hairpin small interference (si)RNA for MR (AAV.MR-shRNA) was constructed and tested ...

journal_title：Gene therapy

authors： Wang X,Skelley L,Cade R,Sun Z

更新日期：2006-07-01 00:00:00

abstract：:In 2012, we reported that 5 out of 11 subjects in a clinical trial (NCT00372320) administering AdhAQP1 to radiation-damaged parotid glands showed increased saliva flow rates and decreased symptoms over the initial 42 days. AdhAQP1 is a first-generation, E1-deleted, replication-defective, serotype 5 adenoviral vector e...

journal_title：Gene therapy

authors： Zheng C,Baum BJ,Liu X,Goldsmith CM,Perez P,Jang SI,Cotrim AP,McCullagh L,Ambudkar IS,Alevizos I

更新日期：2015-09-01 00:00:00

abstract：:Radioiodide concentrating activity in the thyroid, mediated by human Na+/I- symporter (hNIS), provides a mechanism for effective radioiodide treatment for patients who have invasive, recurrent, and metastatic thyroid cancers after total thyroidectomy. In an attempt to develop hNIS gene transfer for radioiodide therapy...

journal_title：Gene therapy

authors： Cho JY,Xing S,Liu X,Buckwalter TL,Hwa L,Sferra TJ,Chiu IM,Jhiang SM

更新日期：2000-05-01 00:00:00

abstract：:Advanced prostate cancer (PC) still remains incurable. Novel immunogene therapy shows promise as treatment strategy that can target both localized and metastasized PC. In this study, we have developed a PC-specific oncolytic adenovirus (Ad-PL-PPT-E1A) armed with fusion gene of prostate-specific antigen and CD40 ligand...

journal_title：Gene therapy

authors： Yang YF,Xue SY,Lu ZZ,Xiao FJ,Yin Y,Zhang QW,Wu CT,Wang H,Wang LS

更新日期：2014-08-01 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：:We have previously synthesized a new cationic liposome that displays high efficiency and low toxicity, 3 beta[l-ornithinamide-carbamoyl] cholesterol (O-Chol), using solid-phase synthesis. In this study, O-Chol was applied to in vitro and in vivo models of ovarian cancer. Intraperitoneal gene delivery for peritoneal di...

journal_title：Gene therapy

authors： Lee MJ,Cho SS,You JR,Lee Y,Kang BD,Choi JS,Park JW,Suh YL,Kim JA,Kim DK,Park JS

更新日期：2002-07-01 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：:The corticospinal tract (CST) is extensively used as a model system for assessing potential therapies to enhance neuronal regeneration and functional recovery following spinal cord injury (SCI). However, efficient transduction of the CST is challenging and remains to be optimised. Recombinant adeno-associated viral (A...

journal_title：Gene therapy

authors： Hutson TH,Verhaagen J,Yáñez-Muñoz RJ,Moon LD

更新日期：2012-01-01 00:00:00

abstract：:Newcastle disease virus (NDV) is an oncolytic paramyxovirus with a nonsegmented single-stranded RNA genome. In this report, a recombinant oncolytic NDV was passaged in human tumor xenografts and reisolated and characterized after two rounds of bioselection. Several isolates could be recovered that differed from the pa...

journal_title：Gene therapy

authors： Beier R,Hermiston T,Mumberg D

更新日期：2013-01-01 00:00:00

abstract：:Parkinson's disease is a prevalent progressive degenerative disorder of the elderly. There is a current need for novel therapeutic strategies because the standard levodopa pharmacotherapy is only temporarily efficacious. Recently, there have been some high-profile successful preclinical results obtained in animal mode...

journal_title：Gene therapy

authors： Manfredsson FP,Lewin AS,Mandel RJ

更新日期：2006-03-01 00:00:00

abstract：:The existence of cells with stem cell-like abilities derived from various tissues can now be extended to include the skeletal muscle compartment. Although researchers have focused on the utilization of these cells with regard to their myogenic capacity, initially exploring more efficient cellular therapy treatments fo...

journal_title：Gene therapy

authors： Jankowski RJ,Deasy BM,Huard J

更新日期：2002-05-01 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：:Integration site analysis was performed on six dogs with canine leukocyte adhesion deficiency (CLAD) that survived greater than 1 year after infusion of autologous CD34+ bone marrow cells transduced with a gammaretroviral vector expressing canine CD18. A total of 387 retroviral insertion sites (RIS) were identified in...

journal_title：Gene therapy

authors： Hai M,Adler RL,Bauer TR Jr,Tuschong LM,Gu YC,Wu X,Hickstein DD

更新日期：2008-07-01 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：:By-pass surgery and percutaneous transluminal (coronary) angioplasty, PT(C)A, are standard techniques for the treatment of vascular occlusions. Their usefulness is limited by by-pass graft failure and restenosis occurring after the procedures. Twenty percent of patients treated with PTCA/PTA need a new revascularizati...

journal_title：Gene therapy

authors： Rutanen J,Puhakka H,Ylä-Herttuala S

更新日期：2002-11-01 00:00:00

abstract：:With the long-term goal of developing a gene-based treatment for osteoarthritis (OA), we performed studies to evaluate the equine joint as a model for adeno-associated virus (AAV)-mediated gene transfer to large, weight-bearing human joints. A self-complementary AAV2 vector containing the coding regions for human inte...

journal_title：Gene therapy

authors： Watson RS,Broome TA,Levings PP,Rice BL,Kay JD,Smith AD,Gouze E,Gouze JN,Dacanay EA,Hauswirth WW,Nickerson DM,Dark MJ,Colahan PT,Ghivizzani SC

更新日期：2013-06-01 00:00:00

abstract：:We demonstrated previously that the additive-type recombinant Sendai virus (rSeV) is highly efficient for use in pulmonary gene transfer; however, rSeV exhibits inflammatory responses. To overcome this problem, we tested newly developed non-transmissible constructs, namely, temperature-sensitive F-deleted vector, rSeV...

journal_title：Gene therapy

authors： Tanaka S,Yonemitsu Y,Yoshida K,Okano S,Kondo H,Inoue M,Hasegawa M,Masumoto K,Suita S,Taguchi T,Sueishi K

更新日期：2007-07-01 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：:A number of genetic disorders are manifested in cutaneous epithelium and gene therapy approaches for treatment of such diseases are being considered. A successful gene therapy protocol requires durable and correctly targeted gene expression within the tissue. The continuous renewal and high levels of compartmentalizat...

journal_title：Gene therapy

authors： Ghazizadeh S,Doumeng C,Taichman LB

更新日期：2002-10-01 00:00:00

abstract：:Angiogenesis plays a pivotal role in tumor growth, tissue invasion and metastasis. Endostatin is an angiogenesis inhibitor and has been shown to reduce tumor growth in animal models. However, therapy with recombinant endostatin protein was hampered by its short half-life and very-low yield of bioactive protein. We per...

journal_title：Gene therapy

authors： Li HL,Li S,Shao JY,Lin XB,Cao Y,Jiang WQ,Liu RY,Zhao P,Zhu XF,Zeng MS,Guan ZZ,Huang W

更新日期：2008-02-01 00:00:00

abstract：:To identify the intracellular barriers to efficient gene transfer, we studied the intracellular trafficking of biotinylated plasmid DNA complexed with either fluorescein-conjugated lactosylated or mannosylated polylysine by confocal microscopy. Both are known to be taken up by cystic fibrosis airway epithelial cells (...

journal_title：Gene therapy

authors： Grosse S,Tremeau-Bravard A,Aron Y,Briand P,Fajac I

更新日期：2002-08-01 00:00:00

abstract：:Bone marrow presents an attractive option for the treatment of articular cartilage defects as it is readily accessible, it contains mesenchymal progenitor cells that can undergo chondrogenic differentiation and, once coagulated, it provides a natural scaffold that contains the cells within the defect. This study was p...

journal_title：Gene therapy

authors： Ivkovic A,Pascher A,Hudetz D,Maticic D,Jelic M,Dickinson S,Loparic M,Haspl M,Windhager R,Pecina M

更新日期：2010-06-01 00:00:00

abstract：:In view of our recent findings that a truncated form of the envelope (Env) glycoprotein of human immunodeficiency virus type 1 (HIV-1) was efficiently incorporated into MoMLV particles, we studied the generation of Moloney murine leukemia virus (MoMLV)/simian immunodeficiency virus (SIV) pseudotypes. Unlike HIV-1, bot...

journal_title：Gene therapy

authors： Indraccolo S,Minuzzo S,Feroli F,Mammano F,Calderazzo F,Chieco-Bianchi L,Amadori A

更新日期：1998-02-01 00:00:00

abstract：:Citrullinemia type 1 (CTLN1) is an autosomal recessive disorder of metabolism caused by a deficiency of argininosuccinate synthetase. Despite optimal management, CTLN1 patients still suffer from lethal metabolic instability and experience life-threatening episodes of acute hyperammonemia. A murine model of CTLN1 (fold...

journal_title：Gene therapy

authors： Chandler RJ,Tarasenko TN,Cusmano-Ozog K,Sun Q,Sutton VR,Venditti CP,McGuire PJ

更新日期：2013-12-01 00:00:00

abstract：:Active disease of tuberculosis (TB) can be developed decades later by either a relapse of the initial infection (endogenous reactivation) or by an entrance of the secondary infection (exogenous reinfection), since the current chemotherapy cannot lead to complete elimination of tuberculosis. Although the immunotherapeu...

journal_title：Gene therapy

authors： Ha SJ,Jeon BY,Youn JI,Kim SC,Cho SN,Sung YC

更新日期：2005-04-01 00:00:00

abstract：:The umbilical cord provides a rich source of primitive mesenchymal stem cells (human umbilical cord mesenchymal stem cells (HUMSCs)), which have the potential for transplantation-based treatments of Parkinson's Disease (PD). Our pervious study indicated that adenovirus-associated virus-mediated intrastriatal delivery ...

journal_title：Gene therapy

authors： Xiong N,Zhang Z,Huang J,Chen C,Zhang Z,Jia M,Xiong J,Liu X,Wang F,Cao X,Liang Z,Sun S,Lin Z,Wang T

更新日期：2011-04-01 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：:We report the discovery, on routine screening, of a replication-competent retrovirus (RCR) produced from a third generation amphotropic packaging cell line, GP + envAM12. In this line, the gag-pol and env helper genes are located on separate plasmids to minimise the chances of recombination events that may lead to RCR...

journal_title：Gene therapy

authors： Chong H,Vile RG

更新日期：1996-07-01 00:00:00

abstract：:Success of gene therapy for diseases affecting the T cell lineage depends on the thymic repopulation by genetically engineered hematopoietic progenitor cells (HPC). Although it has been shown that retrovirally transduced HPC can repopulate the thymus, little information is available on the effect of the culture protoc...

journal_title：Gene therapy

authors： Verhasselt B,Naessens E,De Smedt M,Plum J

更新日期：2000-05-01 00:00:00