Cultured human myoblasts and myotubes show markedly different transducibility by replication-defective adenovirus recombinants.

abstract：:The sodium iodide symporter (NIS) mediates iodide uptake into thyrocytes and is the molecular basis of thyroid radioiodine therapy. We previously have shown that NIS gene transfer into the F98 rat gliomas facilitated tumor imaging and increased survival by radioiodine. In this study, we show that: (1) the therapeutic ...

journal_title：Gene therapy

authors： Shen DH,Marsee DK,Schaap J,Yang W,Cho JY,Hinkle G,Nagaraja HN,Kloos RT,Barth RF,Jhiang SM

更新日期：2004-01-01 00:00:00

abstract：:Foamy virus (FV) vectors are promising for hematopoietic stem cell (HSC) gene therapy but preclinical data on the clonal composition of FV vector-transduced human repopulating cells is needed. Human CD34(+) human cord blood cells were transduced with an FV vector encoding a methylguanine methyltransferase (MGMT)P140K ...

journal_title：Gene therapy

authors： Olszko ME,Adair JE,Linde I,Rae DT,Trobridge P,Hocum JD,Rawlings DJ,Kiem HP,Trobridge GD

更新日期：2015-07-01 00:00:00

abstract：:Acute lung injury (ALI) and acute respiratory distress syndrome (ARDS) are associated with diverse disorders and characterized by disruption of the alveolar-capillary barrier, leakage of edema fluid into the lung, and substantial inflammation leading to acute respiratory failure. Gene therapy is a potentially powerful...

journal_title：Gene therapy

authors： Lin X,Barravecchia M,Kothari P,Young JL,Dean DA

更新日期：2016-06-01 00:00:00

abstract：:Recombinant Autographa californica multiple nuclear polyhedrosis viruses (AcMNPV) have recently been shown to transduce mammalian cells in vitro. Since baculoviruses offer many advantages over viruses currently used in gene therapy, we have tested them for in vivo gene transfer by constructing a baculovirus bearing a ...

journal_title：Gene therapy

authors： Airenne KJ,Hiltunen MO,Turunen MP,Turunen AM,Laitinen OH,Kulomaa MS,Ylä-Herttuala S

更新日期：2000-09-01 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：:In the last two decades, remarkable advances have been made in the development of technologies used to engineer new aptamers and ribozymes. This has encouraged interest among researchers who seek to create new types of gene-control systems that can be made to respond specifically to small-molecule signals. Validation ...

journal_title：Gene therapy

authors： Link KH,Breaker RR

更新日期：2009-10-01 00:00:00

abstract：:Insulin-like growth factor-I (IGF-I) and its receptor (IGF-IR) are essential for normal ocular development and are expressed in numerous ocular cell types including lens epithelial cells, retinal pigment epithelial cells, Müller cells and endothelial cells. Endothelial cell proliferation is a common feature of prolife...

journal_title：Gene therapy

authors： Shaw LC,Pan H,Afzal A,Calzi SL,Spoerri PE,Sullivan SM,Grant MB

更新日期：2006-05-01 00:00:00

abstract：:We report on systemic delivery and long-term biological effects of apolipoprotein E (apoE) obtained by intramuscular (i.m.) plasmid DNA injection. ApoE plays an important role in lipoprotein catabolism and apoE knock-out mice develop severe hypercholesterolemia and diffuse atherosclerosis. We have injected apoE-defici...

journal_title：Gene therapy

authors： Rinaldi M,Catapano AL,Parrella P,Ciafrè SA,Signori E,Seripa D,Uboldi P,Antonini R,Ricci G,Farace MG,Fazio VM

更新日期：2000-11-01 00:00:00

abstract：:For successful cancer gene therapy via intravenous (i.v.) administration, it is essential to optimize the stability of carriers in the systemic circulation and the cellular association after the accumulation of the carrier in tumor tissue. However, a dilemma exists regarding the use of poly(ethylene glycol) (PEG), whi...

journal_title：Gene therapy

authors： Hatakeyama H,Akita H,Kogure K,Oishi M,Nagasaki Y,Kihira Y,Ueno M,Kobayashi H,Kikuchi H,Harashima H

更新日期：2007-01-01 00:00:00

abstract：:Current tools for the inhibition of microRNA (miR) function are limited to modified antisense oligonucleotides, sponges and decoy RNA molecules and none have been used to understand miR function during development. CRISPR/Cas-mediated deletion of miR sequences within the genome requires multiple chromosomal deletions ...

journal_title：Gene therapy

authors： Cao H,Yu W,Li X,Wang J,Gao S,Holton NE,Eliason S,Sharp T,Amendt BA

更新日期：2016-06-01 00:00:00

abstract：:The efficiency of plasmid gene transfer to skeletal muscle can be significantly improved by the application of an electrical field to the muscle following injection of plasmid DNA. However, this electrotransfer is associated with significant muscle damage which may result in substantial loss of transfected muscle fibr...

journal_title：Gene therapy

authors： McMahon JM,Signori E,Wells KE,Fazio VM,Wells DJ

更新日期：2001-08-01 00:00:00

abstract：:Various methods for determining the expression of the beta-galactosidase (beta-gal) gene after retroviral transduction were compared as a means to assess retroviral titre. To allow better comparison, different retroviral vectors were constructed carrying two mutants of the green fluorescent protein and assessed as sen...

journal_title：Gene therapy

authors： Klein D,Indraccolo S,von Rombs K,Amadori A,Salmons B,Günzburg WH

更新日期：1997-11-01 00:00:00

abstract：:The CMV promoter drives high transgene expression and is one of the most commonly used promoters for gene transfer. Tissue-specific mammalian promoters provide an alternative, and it would be useful to have a system to directly compare them to viral promoters free from potential confounding vector-related effects. In ...

journal_title：Gene therapy

authors： Kachi S,Esumi N,Zack DJ,Campochiaro PA

更新日期：2006-05-01 00:00:00

abstract：:Dendritic cell (DC)-based vaccine approaches are being actively evaluated for developing immunotherapeutic agents against cancers. In this study, we investigated the use of engineered DCs expressing transgenic tumor-associated antigen hgp100 and the regulatory cytokine interleukin-21, namely DC-hgp100/mIL-21, as a the...

journal_title：Gene therapy

authors： Aravindaram K,Wang PH,Yin SY,Yang NS

更新日期：2014-05-01 00:00:00

abstract：:Retroviral vectors have become the primary tool for gene delivery into hematopoietic cells, including T lymphocytes. Lentiviral vectors offer an advantage over Moloney murine leukemia virus (MuLV) vectors because of their ability to translocate across an intact nuclear membrane and integrate into the genome of nonprol...

journal_title：Gene therapy

authors： Dardalhon V,Herpers B,Noraz N,Pflumio F,Guetard D,Leveau C,Dubart-Kupperschmitt A,Charneau P,Taylor N

更新日期：2001-02-01 00:00:00

abstract：:The potential of gene therapy for treatment of lung disease remains unrealised. Early model systems often resulted in promising efficiency of gene transfer, only to prove irreproducible in the clinic. While problems such as induction of host immune responses and duration of expression also need to be addressed, it is ...

journal_title：Gene therapy

authors： McBride S,Rannie D,Harrison DJ

更新日期：2000-04-01 00:00:00

abstract：:To investigate to what extent myeloablation, graft size, and ex vivo manipulation influence the engraftment and long-term survival of transduced murine hematopoietic cells, groups of C57BL/6J (CD45.2) mice receiving total body irradiation (TBI) (1-9 Gy) or no irradiation were transplanted with either transduced bone m...

journal_title：Gene therapy

authors： Puig T,Kádár E,Limón A,Cancelas JA,Eixarch H,Luquín L,García M,Barquinero J

更新日期：2002-11-01 00:00:00

abstract：:The expression of naked plasmid DNA coding for firefly luciferase (pRSVluc) or a secreted protein, human-alpha-1-antitrypsin (pRcCMVhAAT) in mouse skeletal muscle was characterized following administration by an improved intramuscular injection technique. Injection guided by intense illumination along the longitudinal...

journal_title：Gene therapy

authors： Levy MY,Barron LG,Meyer KB,Szoka FC Jr

更新日期：1996-03-01 00:00:00

abstract：:Chimeric oligonucleotides have been used successfully to correct point and frameshift mutations in several cell types, as well as in animal and plant models. However, their application to primitive human blood cells has been limited. In this investigation, chimeric oligonucleotides designed to direct a site-specific n...

journal_title：Gene therapy

authors： Liu H,Agarwal S,Kmiec E,Davis BR

更新日期：2002-01-01 00:00:00

abstract：:We evaluated the efficacy of equine infectious anaemia virus (EIAV)-based lentiviral vectors encoding endostatin (EIAV.endostatin) or angiostatin (EIAV.angiostatin) in inhibiting angiogenesis and vascular hyperpermeability in the laser-induced model of choroidal neovascularisation (CNV). Equine infectious anaemia viru...

journal_title：Gene therapy

authors： Balaggan KS,Binley K,Esapa M,MacLaren RE,Iqball S,Duran Y,Pearson RA,Kan O,Barker SE,Smith AJ,Bainbridge JW,Naylor S,Ali RR

更新日期：2006-08-01 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：:The recent surge of DNA sequence information resulting from the efforts of agencies interested in deciphering the human genetic code has facilitated technological developments that have been critical in the identification of genes associated with numerous disease pathologies. In addition, these efforts have opened the...

journal_title：Gene therapy

authors： Gruenert DC,Novelli G,Dallapiccola B,Colosimo A

更新日期：2002-06-01 00:00:00

abstract：:Intramuscular (i.m.) injection of a plasmid encoding human carcinoembryonic antigen (CEA) elicited immunity against transplanted syngeneic (C57BL/6) CEA-positive Lewis lung carcinoma (CEA/LLC) cells, but tumors still appeared in all mice. In wild-type mice, coinjection of an IL-12 plasmid markedly enhanced anti-CEA hu...

journal_title：Gene therapy

authors： Song K,Chang Y,Prud'homme GJ

更新日期：2000-09-01 00:00:00

abstract：:Solid tumors meet their demands for nascent blood vessels and increased glycolysis, to combat hypoxia, by activating multiple genes involved in angiogenesis and glucose metabolism. Hypoxia inducible factor-1 (HIF-1) is a constitutively expressed basic helix-loop-helix transcription factor, formed by the assembly of HI...

journal_title：Gene therapy

authors： Sun X,Kanwar JR,Leung E,Lehnert K,Wang D,Krissansen GW

更新日期：2001-04-01 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：:Despite significant advances in basic research, the treatment of degenerative diseases of the nervous system remains one of the greatest challenges for translational medicine. The childhood onset motor neuron disorder spinal muscular atrophy (SMA) has been viewed as one of the more tractable targets for molecular ther...

journal_title：Gene therapy

authors： Talbot K,Tizzano EF

更新日期：2017-09-01 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：:The successful development of adenovirus vectors for vaccines and gene therapy will require a better understanding of the host immune response. Using the ELISPOT assay to measure IFN-gamma-secreting CD8(+) cells, we identify immunodominant epitopes of the adenovirus hexon and DNA-binding protein in BALB/c and C57BL/6 ...

journal_title：Gene therapy

authors： McKelvey T,Tang A,Bett AJ,Casimiro DR,Chastain M

更新日期：2004-05-01 00:00:00

abstract：:The number of clinical trials using gene transfer technology, either active or under discussion, is increasing rapidly. However, little information is available describing the regulatory procedures or safety specifications that must be considered before initiation of such trials in Europe. We describe the procedure us...

journal_title：Gene therapy

authors： Caplen NJ,Gao X,Hayes P,Elaswarapu R,Fisher G,Kinrade E,Chakera A,Schorr J,Hughes B,Dorin JR

更新日期：1994-03-01 00:00:00

abstract：:Epstein-Barr virus (EBV)-associated B-cell lymphoproliferative disease (LPD) after hematopoietic stem cell or solid organ transplantation remains a life-threatening complication. Expression of the virus-encoded gene product, EBER, has been shown to prevent apoptosis via blockade of PKR activation. As PKR is a major ce...

journal_title：Gene therapy

authors： Wang PY,Currier MA,Hansford L,Kaplan D,Chiocca EA,Uchida H,Goins WF,Cohen JB,Glorioso JC,van Kuppevelt TH,Mo X,Cripe TP

更新日期：2013-07-01 00:00:00