Intraoperative multiplane transesophageal echocardiography for guiding direct myocardial gene transfer of vascular endothelial growth factor in patients with refractory angina pectoris.

abstract：:Oncolytic measles virus (MV) encoding the human thyroidal sodium iodide symporter (MV-NIS) has proved to be safe after intraperitoneal or intravenous administration in patients with ovarian cancer or multiple myeloma, respectively, but it has not yet been administered through intratumoral injection in humans. Squamous...

journal_title：Human gene therapy

authors： Li H,Peng KW,Russell SJ

更新日期：2012-03-01 00:00:00

abstract：:Deficiency of glycogen branching enzyme (GBE) causes glycogen storage disease type IV (GSD IV), which is characterized by the accumulation of a less branched, poorly soluble form of glycogen called polyglucosan (PG) in multiple tissues. This study evaluates the efficacy of gene therapy with an adeno-associated viral (...

journal_title：Human gene therapy

authors： Yi H,Zhang Q,Brooks ED,Yang C,Thurberg BL,Kishnani PS,Sun B

更新日期：2017-03-01 00:00:00

abstract：:MDA-MB-231, an HLA-A2(+), HER2/neu(+) allogeneic breast cancer cell line genetically modified to express the costimulatory molecule CD80 (B7-1), was used to vaccinate 30 women with previously treated stage IV breast cancer. Expression of CD80 conferred the ability to deliver a costimulatory signal and thereby improved...

journal_title：Human gene therapy

authors： Dols A,Smith JW 2nd,Meijer SL,Fox BA,Hu HM,Walker E,Rosenheim S,Moudgil T,Doran T,Wood W,Seligman M,Alvord WG,Schoof D,Urba WJ

更新日期：2003-07-20 00:00:00

abstract：:Successful gene therapy approaches for metachromatic leukodystrophy (MLD), based either on hematopoietic stem/progenitor cells (HSPCs) or direct central nervous system (CNS) gene transfer, highlighted a requirement for high levels of arylsulfatase A (ARSA) expression to achieve correction of disease manifestations in ...

journal_title：Human gene therapy

authors： Capotondo A,Cesani M,Pepe S,Fasano S,Gregori S,Tononi L,Venneri MA,Brambilla R,Quattrini A,Ballabio A,Cosma MP,Naldini L,Biffi A

更新日期：2007-09-01 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 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：:Peritoneal compartmentalization of advanced stage ovarian cancer provides a rational scenario for gene therapy strategies. Several groups are exploring intraperitoneal administration of adenoviral (Ad) vectors for this purpose. We examined in vitro gene transfer in the presence of ascites fluid from ovarian cancer pat...

journal_title：Human gene therapy

authors： Blackwell JL,Li H,Gomez-Navarro J,Dmitriev I,Krasnykh V,Richter CA,Shaw DR,Alvarez RD,Curiel DT,Strong TV

更新日期：2000-08-10 00:00:00

abstract：:If established cultured cell lines genetically modified to secrete desired gene products could be implanted in different allogeneic recipients without immune rejection, novel gene products would be delivered more cost effectively. We tested this strategy by encapsulating mouse Ltk- cells transfected with the human gro...

journal_title：Human gene therapy

authors： Chang PL,Shen N,Westcott AJ

更新日期：1993-08-01 00:00:00

abstract：:Vectors based on lentiviruses have become potent tools for efficient gene transfer to multiple cell types both in vitro and in vivo. In part this is attributable to the stability of transduction afforded by integration into the target cell genome. However, evidence indicates that episomal forms of the vector can also ...

journal_title：Human gene therapy

authors： Philpott NJ,Thrasher AJ

更新日期：2007-06-01 00:00:00

abstract：:This multicenter phase I/II study evaluated the safety, pharmacokinetics, and antitumor effects of repeated doses of NV1020, a genetically engineered oncolytic herpes simplex virus, in patients with advanced metastatic colorectal cancer (mCRC). Patients with liver-dominant mCRC received four fixed NV1020 doses via wee...

journal_title：Human gene therapy

authors： Geevarghese SK,Geller DA,de Haan HA,Hörer M,Knoll AE,Mescheder A,Nemunaitis J,Reid TR,Sze DY,Tanabe KK,Tawfik H

更新日期：2010-09-01 00:00:00

abstract：:Duchenne muscular dystrophy (DMD) and other inherited myopathies lead to progressive destruction of most skeletal muscles in the body, including those responsible for maintaining respiration. DMD is a fatal disorder caused by defects in the dystrophin gene. Recombinant adenovirus vectors (AdV) are considered a promisi...

journal_title：Human gene therapy

authors： Cho WK,Ebihara S,Nalbantoglu J,Gilbert R,Massie B,Holland P,Karpati G,Petrof BJ

更新日期：2000-03-20 00:00:00

abstract：:Adenoviruses are attractive vectors for gene transfer into cardiac muscle. However, their promiscuous tissue tropism, which leads to an ectopic expression of the transgene, is a considerable limitation. To restrict expression to cardiomyocytes, we have constructed two recombinant adenoviruses (Ad-MLC2-250betagal and A...

journal_title：Human gene therapy

authors： Griscelli F,Gilardi-Hebenstreit P,Hanania N,Franz WM,Opolon P,Perricaudet M,Ragot T

更新日期：1998-09-01 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：:Point mutations in the dystrophin gene cause dystrophin deficiency and muscular dystrophy in the mdx mouse and a subset of patients with Duchenne muscular dystrophy. As an approach to gene therapy for muscular dystrophies due to point mutations, we have studied the ability of RNA-DNA chimeric oligonucleotides (chimera...

journal_title：Human gene therapy

authors： Bertoni C,Rando TA

更新日期：2002-04-10 00:00:00

abstract：:Clinical applications of gene therapy require advances in gene delivery systems. Although numerous clinical trials are already underway, the ultimate success of gene therapies will depend on gene transfer vectors that facilitate the expression of a specific gene at therapeutic levels in the desired cell populations wi...

journal_title：Human gene therapy

authors： Anderson DM,Hall LL,Ayyalapu AR,Irion VR,Nantz MH,Hecker JG

更新日期：2003-02-10 00:00:00

abstract：:Gene therapy for hemophilia B has been shown to result in long-term expression and immune tolerance to factor IX (F.IX) after in vivo transduction of hepatocytes with adeno-associated viral (AAV-2) vectors in experimental animals. An optimized protocol was effective in several strains of mice with a factor 9 gene dele...

journal_title：Human gene therapy

authors： Cooper M,Nayak S,Hoffman BE,Terhorst C,Cao O,Herzog RW

更新日期：2009-07-01 00:00:00

abstract：:Stem-cell therapy is a promising method for treating patients with a wide range of diseases and injuries. Increasing government funding of scientific research has promoted rapid developments in stem-cell research in China, as evidenced by the substantial increase in the number and quality of publications in the past 5...

journal_title：Human gene therapy

authors： Hu L,Zhao B,Wang S

更新日期：2018-02-01 00:00:00

abstract：:In vivo electroporation of plasmid DNA (DNA-EP) is an efficient and safe method for vaccines. It results in increased DNA uptake, enhances protein expression, and augments immune responses to the target antigen in a variety of species. To further improve the efficacy of DNA-EP, we evaluated small interfering RNA (siRN...

journal_title：Human gene therapy

authors： Dharmapuri S,Aurisicchio L,Biondo A,Welsh N,Ciliberto G,La Monica N

更新日期：2009-06-01 00:00:00

abstract：:Antitumor gene therapy using herpes simplex type 1 thymidine kinase (TKh) and ganciclovir (GCV) treatment has revealed an important intratumoral bystander effect. A whole tumor can be eliminated when only a fraction of its tumor cells express TKh. We now report that the bystander effect not only acts within a tumor, b...

journal_title：Human gene therapy

authors： Kianmanesh AR,Perrin H,Panis Y,Fabre M,Nagy HJ,Houssin D,Klatzmann D

更新日期：1997-10-10 00:00:00

abstract：:The management of disorders of the nervous system remains a medical challenge. The key goals are to understand disease mechanisms, to validate therapeutic targets, and to develop new therapeutic strategies. Viral vector-mediated gene transfer can meet these goals and vectors based on lentiviruses have particularly use...

journal_title：Human gene therapy

authors： Wong LF,Goodhead L,Prat C,Mitrophanous KA,Kingsman SM,Mazarakis ND

更新日期：2006-01-01 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：:At present, much more studies have focused on the role of microRNAs in osteoporosis, but the more specific role of microRNA-150-3p (miR-150-3p) in osteoporosis still needs full exploration. We aim at investigating the role of miR-150-3p in osteoporosis and at exploring the related mechanisms. Bone marrow mesenchymal s...

journal_title：Human gene therapy

authors： Qiu M,Zhai S,Fu Q,Liu D

更新日期：2021-01-22 00:00:00

abstract：:Liver ischemia-reperfusion (I/R) injury is a multifactorial process that affects graft function after liver transplantation. Inflammatory cytokines, such as tumor necrosis factor (TNF)-α, interleukin (IL)-6, IL-1β, and IL-18, have been shown to play key roles in the pathophysiology of liver I/R injury. Studies have in...

journal_title：Human gene therapy

authors： Zhu P,Duan L,Chen J,Xiong A,Xu Q,Zhang H,Zheng F,Tan Z,Gong F,Fang M

更新日期：2011-07-01 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 T cell co-stimulatory molecule B7-1 was transduced into a poorly immunogenic murine neuroblastoma cell line (Neuro-2a, N-2a) alone or in combination with MHC class II genes to test the ability of these genes to stimulate antitumor immunity. N-2a cells transduced with B7-1 exhibited reduced tumorigenicity, whereas ...

journal_title：Human gene therapy

authors： Heuer JG,Tucker-McClung C,Gonin R,Hock RA

更新日期：1996-11-10 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：: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：:Atrogin-1 or muscle atrophy F-box (MAFbx) is a major atrophy-related E3 ubiquitin ligase highly expressed in skeletal muscle during muscle atrophy and other disease states such as sepsis, cancer cachexia, and fasting. In this paper, we report experiments inhibiting MAFbx activity in fasting mice and in the skeletal my...

journal_title：Human gene therapy

authors： Cong H,Sun L,Liu C,Tien P

更新日期：2011-03-01 00:00:00

abstract：:Foamy virus (FV) vectors are a promising gene delivery system for use in hematopoietic stem cell gene therapy. Previous FV vector marking studies in the NOD/SCID xenotransplantation model used umbilical cord blood (UCB)-derived SCID repopulating cells (SRCs) that were assayed 5-10 weeks posttransplantation. We now rep...

journal_title：Human gene therapy

authors： Josephson NC,Trobridge G,Russell DW

更新日期：2004-01-01 00:00:00

abstract：:Conditionally replicative adenovirus (CRAd) vectors are designed for specific oncolytic replication in tumor tissues with concomitant sparing of normal cells. As such, CRAds offer an unprecedented level of anticancer potential for malignancies that have been refractory to previous cancer gene therapy interventions. CR...

journal_title：Human gene therapy

authors： Li H,Haviv YS,Derdeyn CA,Lam J,Coolidge C,Hunter E,Curiel DT,Blackwell JL

更新日期：2001-12-10 00:00:00