Recombinant truncated dystrophin minigenes: construction, expression, and adenoviral delivery.

abstract：:Targeted integration into a genomic safe harbor, such as the AAVS1 locus on chromosome 19, promises predictable transgene expression and reduces the risk of insertional mutagenesis in the host genome. The application of gamma-retroviral long terminal repeat (LTR)-driven vectors, which semirandomly integrate into the g...

journal_title：Human gene therapy

authors： Klatt D,Cheng E,Hoffmann D,Santilli G,Thrasher AJ,Brendel C,Schambach A

更新日期：2020-02-01 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：:Glycogen storage disease type II (GSD-II) is a lethal, autosomal recessive metabolic myopathy caused by a lack of acid-alpha-glucosidase (GAA) activity in the cardiac and skeletal muscles. Absence of adequate intralysosomal GAA activity results in massive amounts of glycogen accumulation in multiple muscle groups, res...

journal_title：Human gene therapy

authors： Ding EY,Hodges BL,Hu H,McVie-Wylie AJ,Serra D,Migone FK,Pressley D,Chen YT,Amalfitano A

更新日期：2001-05-20 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

abstract：:The use of viral thymidine kinase (TK) gene coupled with the administration of ganciclovir to render cancer cell death has been studied extensively. Many of these experiments utilized retrovirus to transfer the TK gene under the control of a nonspecific promoter. Because nonspecific expression of the viral TK gene may...

journal_title：Human gene therapy

authors： Su H,Chang JC,Xu SM,Kan YW

更新日期：1996-03-01 00:00:00

abstract：:The epithelial-mesenchymal transition (EMT) has been recognized to occur during embryonic development, fibrosis, and tumor metastasis. Nuclear factor (NF)-κB plays a central role in mediating the inflammation and wound-healing responses during liver fibrogenesis. However, the involvement of NF-κB during EMT in liver c...

journal_title：Human gene therapy

authors： Kim KH,Lee WR,Kang YN,Chang YC,Park KK

更新日期：2014-08-01 00:00:00

abstract：:Bone marrow mesenchymal stem cells (BMSCs) represent an important source of cells for tissue repair. The tropism of these cells to the sites of injury and tumors has been well established. Their tumor-homing properties make BMSCs good candidates as antitumor agent delivery vehicles. In this study, we showed that BMSCs...

journal_title：Human gene therapy

authors： Song C,Xiang J,Tang J,Hirst DG,Zhou J,Chan KM,Li G

更新日期：2011-04-01 00:00:00

abstract：:The initial stages following the in vitro cytokine stimulation of human cord blood CD34+ cells overlap with the period when lentiviral gene transfer is typically performed. Single-cell transcriptional profiling and time-lapse microscopy were used to investigate how the vector-cell crosstalk impacts on the fate decisio...

journal_title：Human gene therapy

authors： Moussy A,Papili Gao N,Corre G,Poletti V,Majdoul S,Fenard D,Gunawan R,Stockholm D,Páldi A

更新日期：2019-08-01 00:00:00

abstract：:Despite efforts toward improvements in retrovirus-mediated gene transfer, stable high-level expression of a therapeutic gene in human hematopoietic stem cells remains a great challenge. We have evaluated the efficiency of different viral long terminal repeats (LTRs) in long-term expression of a transgene in vivo, usin...

journal_title：Human gene therapy

authors： Kaneko S,Onodera M,Fujiki Y,Nagasawa T,Nakauchi H

更新日期：2001-01-01 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：: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：:Immunotherapy with whole cell cancer vaccines has been tested in various tumor types. This study investigated the safety profile and antitumor activity of an allogeneic prostate carcinoma cell line, LNCaP, expressing recombinant human interleukin-2 and human interferon-gamma. Thirty HLA-A*0201-matched patients with pr...

journal_title：Human gene therapy

authors： Brill TH,Kübler HR,Pohla H,Buchner A,Fend F,Schuster T,van Randenborgh H,Paul R,Kummer T,Plank C,Eisele B,Breul J,Hartung R,Schendel DJ,Gansbacher B

更新日期：2009-12-01 00:00:00

abstract：:Administration of plasmid/lipid complexes to the lung airways for the treatment of metastatic pulmonary diseases represents a new strategy of gene therapy. In this study we present evidence that intratracheal administration of a plasmid encoding murine IL-12 complexed with N-[1-(2,3-dioleyloxy)propyl)-N,N,N-trimethyla...

journal_title：Human gene therapy

authors： Blezinger P,Freimark BD,Matar M,Wilson E,Singhal A,Min W,Nordstrom JL,Pericle F

更新日期：1999-03-20 00:00:00

abstract：:Retroviral vectors encoding glucose-responsive promoters driving furin expression may provide an amplified, glucose-regulated secretion of insulin. We constructed LhI*TFSN virus to encode a glucose-regulatable transforming growth factor alpha promoter controlling furin expression with a viral LTR promoter driving cons...

journal_title：Human gene therapy

authors： Barry SC,Ramesh N,Lejnieks D,Simonson WT,Kemper L,Lernmark A,Osborne WR

更新日期：2001-01-20 00:00:00

abstract：:Transfer of a herpes simplex virus-derived thymidine kinase (HSV-tk) gene into brain tumor cells and subsequent ganciclovir (GCV) treatment has been shown by others to be an effective treatment in rats with intracerebrally inoculated 9L gliosarcomas. Mechanism of action and reproducibility are, however, still a matter...

journal_title：Human gene therapy

authors： Vincent AJ,Vogels R,Someren GV,Esandi MC,Noteboom JL,Avezaat CJ,Vecht C,Bekkum DW,Valerio D,Bout A,Hoogerbrugge PM

更新日期：1996-01-20 00:00:00

abstract：:Targeted delivery of intravenously administered genetically altered cells or stem cells is still in an early stage of investigation. We developed a method of delivering iron oxide (ferumoxide)-labeled mesenchymal stem cells (MSCs) to a targeted area in an animal model by applying an external magnet. Rats with or witho...

journal_title：Human gene therapy

authors： Arbab AS,Jordan EK,Wilson LB,Yocum GT,Lewis BK,Frank JA

更新日期：2004-04-01 00:00:00

abstract：:Human adenoviruses are the most widely used vectors in gene medicine, with applications ranging from oncolytic therapies to vaccinations, but adenovirus vectors are not without side effects. In addition, natural adenoviruses pose severe risks for immunocompromised people, yet infections are usually mild and self-limit...

journal_title：Human gene therapy

authors： Hendrickx R,Stichling N,Koelen J,Kuryk L,Lipiec A,Greber UF

更新日期：2014-04-01 00:00:00

abstract：:Stem cell mobilization to injured tissue contributes to neovascularization, resulting in regeneration after myocardial infarction (MI). We previously showed that direct cardiac injection of a recombinant lentivirus (LV) that engineers expression of membrane-bound stem cell factor (mSCF) improves outcomes immediately a...

journal_title：Human gene therapy

authors： Sun Z,Lee CJ,Mejia-Guerrero S,Zhang Y,Higuchi K,Li RK,Medin JA

更新日期：2012-12-01 00:00:00

abstract：:Fabry disease, caused by a deficiency of lysosomal enzyme alpha-galactosidase A (alpha-gal A), is one of the inherited disorders potentially treatable by gene transfer to hematopoietic stem cells. In this study, a high-titer amphotropic retroviral producer cell line, MFG-alpha-gal A, was established. CD34+ cells from ...

journal_title：Human gene therapy

authors： Takiyama N,Dunigan JT,Vallor MJ,Kase R,Sakuraba H,Barranger JA

更新日期：1999-12-10 00:00:00

abstract：:Virotherapy is a unique modality for the treatment of cancer with oncolytic viruses (OVs) that selectively infect and lyse tumor cells, spread within tumors, and activate anti-tumor immunity. Various viruses are being developed as OVs preclinically and clinically, several of them engineered to encode therapeutic prote...

journal_title：Human gene therapy

authors： Ungerechts G,Engeland CE,Buchholz CJ,Eberle J,Fechner H,Geletneky K,Holm PS,Kreppel F,Kühnel F,Lang KS,Leber MF,Marchini A,Moehler M,Mühlebach MD,Rommelaere J,Springfeld C,Lauer UM,Nettelbeck DM

更新日期：2017-10-01 00:00:00

abstract：:In previous studies we demonstrated that a modified human HSP70b promoter (HSE.70b) directs high levels of gene expression to tumor cells after mild hyperthermia treatment in the range of 41.5-44 degrees C. This transcriptional targeting system exhibits low basal activity at 37 degrees C, is highly induced (950-fold) ...

journal_title：Human gene therapy

authors： Brade AM,Szmitko P,Ngo D,Liu FF,Klamut HJ

更新日期：2003-03-20 00:00:00

abstract：:Glioblastomas account for approximately 20% of all primary brain tumors in adults. Glioblastoma multiforme (GBM) is a highly malignant tumor. In spite of advances in surgery, chemotherapy, and radiotherapy, the life expectancy of the patient with glioblastoma is approximately 11 months. To enhance glioma-specific gene...

journal_title：Human gene therapy

authors： Ho IA,Lam PY,Hui KM

更新日期：2004-08-01 00:00:00

abstract：:The large amounts of recombinant adeno-associated virus (rAAV) vector needed for clinical trials and eventual commercialization require robust, economical, reproducible, and scalable production processes compatible with current good manufacturing practice. rAAV produced using baculovirus and insect cells satisfies the...

journal_title：Human gene therapy

authors： Cecchini S,Virag T,Kotin RM

更新日期：2011-08-01 00:00:00

abstract：:RNA interference (RNAi) is an evolutionarily conserved mechanism of posttranscriptional gene-specific silencing. For in vivo applications, RNAi has been hampered until recently by inefficient delivery methods and by the transient nature of the gene suppression. Lentiviral vectors (LVs) hold great promise for gene ther...

journal_title：Human gene therapy

authors： Van den Haute C,Eggermont K,Nuttin B,Debyser Z,Baekelandt V

更新日期：2003-12-10 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：:Malignant mesothelioma is a tumor of the pleura for which there is no satisfactory treatment. It is almost universally fatal, regardless of the stage of the tumor at the time of diagnosis. Current treatment modalities include surgery, chemotherapy, and radiation therapy, although in some series none of these modalitie...

journal_title：Human gene therapy

authors： Schwarzenberger P,Harrison L,Weinacker A,Marrogi A,Byrne P,Ramesh R,Theodossiou C,Gaumer R,Summer W,Freeman SM,Kolls JK

更新日期：1998-11-20 00:00:00

abstract：:Lung disease associated with disorders such as cystic fibrosis (CF) may be amenable to somatic gene therapy in which there is delivery of the normal gene directly to the respiratory epithelium using E1a- adenovirus (Ad) type 2- or 5-based vectors. For safety reasons, the Ad vectors are rendered replication deficient b...

journal_title：Human gene therapy

authors： Eissa NT,Chu CS,Danel C,Crystal RG

更新日期：1994-09-01 00:00:00

abstract：:Ornithine transcarbamylase deficiency (OTCD) is an inborn error of urea synthesis that has been considered as a model for liver-directed gene therapy. Current treatment has failed to avert a high mortality or morbidity from hyperammonemic coma. Restoration of enzyme activity in the liver should suffice to normalize me...

journal_title：Human gene therapy

authors： Raper SE,Yudkoff M,Chirmule N,Gao GP,Nunes F,Haskal ZJ,Furth EE,Propert KJ,Robinson MB,Magosin S,Simoes H,Speicher L,Hughes J,Tazelaar J,Wivel NA,Wilson JM,Batshaw ML

更新日期：2002-01-01 00:00:00

abstract：:Human hematopoietic stem cells (HSCs) are poorly transduced by vectors based on adenovirus serotype 5 (Ad5). This is primarily due to the paucity of the coxsackievirus-Ad receptor on these cells. In an attempt to change the tropism of Ad5, we constructed a series of chimeric E1-deleted Ad5 vectors in which the shaft a...

journal_title：Human gene therapy

authors： Knaän-Shanzer S,Van Der Velde I,Havenga MJ,Lemckert AA,De Vries AA,Valerio D

更新日期：2001-11-01 00:00:00

abstract：:Cell therapies are treatments in which stem or progenitor cells are stimulated to differentiate into specialized cells able to home to and repair damaged tissues. After their discovery, endothelial progenitor cells (EPCs) stimulated worldwide interest as possible vehicles to perform autologous cell therapy of tumors. ...

journal_title：Human gene therapy

authors： Laurenzana A,Margheri F,Chillà A,Biagioni A,Margheri G,Calorini L,Fibbi G,Del Rosso M

更新日期：2016-10-01 00:00:00