Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell-Derived RPE and Photoreceptor Cells.

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：:DNA-based vaccines able to induce efficient cytotoxic T-cell responses targeting conserved elements (CE) of human immunodeficiency virus type 1 (HIV-1) Gag have been developed. These CE were selected by stringent conservation, the ability to induce T-cell responses with broad human leukocyte antigen coverage, and the ...

journal_title：Human gene therapy

authors： Hu X,Valentin A,Cai Y,Dayton F,Rosati M,Ramírez-Salazar EG,Kulkarni V,Broderick KE,Sardesai NY,Wyatt LS,Earl PL,Moss B,Mullins JI,Pavlakis GN,Felber BK

更新日期：2018-09-01 00:00:00

abstract：:Recombinant adeno-associated virus 2 (rAAV2) has been extensively used as a gene delivery vector for the nervous system. It targets primarily neurons in the nervous system and results in sustained long-term expression of transgenes. New rAAV serotypes have been characterized and demonstrated to have improved transduct...

journal_title：Human gene therapy

authors： Burger C,Nash K,Mandel RJ

更新日期：2005-07-01 00:00:00

abstract：:The central nervous system (CNS) is a predominant site of involvement in several lysosomal storage diseases (LSDs); and for many patients, these diseases are diagnosed only after the onset of symptoms related to the progressive accumulation of macromolecules within lysosomes. The mucopolysaccharidosis type VII (MPS VI...

journal_title：Human gene therapy

authors： Sferra TJ,Qu G,McNeely D,Rennard R,Clark KR,Lo WD,Johnson PR

更新日期：2000-03-01 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：:Newcastle disease virus (NDV) is a naturally oncolytic virus that has been shown to be safe and effective for cancer therapy. Tumor virotherapy using NDV emerged in the 1950s and has advanced more recently by the increased availability of reverse genetics technology. In this study, we constructed a reverse genetics sy...

journal_title：Human gene therapy

authors： Wei D,Sun N,Nan G,Wang Y,Liu HQ,Peeters B,Chen ZN,Bian H

更新日期：2012-07-01 00:00:00

abstract：:Adenovirus (Ad) vectors used for gene therapy are efficient in entering the infected cell and targeting their genome to the nucleus. To study the mechanism of the interaction between Ad and the nuclear envelope we have established an in vitro assay using rat liver nuclei incubated with serotype 5 Ad vector. Binding of...

journal_title：Human gene therapy

authors： Wisnivesky JP,Leopold PL,Crystal RG

更新日期：1999-09-01 00:00:00

abstract：:Umbilical cord blood (CB) from the early gestational human fetus is recognized as a rich source of hematopoietic stem cells. To examine the value of fetal CB for gene therapy of inborn immunohematopoietic disorders, we tested the feasibility of genetic modification of CD34(+) cells from CB at weeks 24 to 34 of pregnan...

journal_title：Human gene therapy

authors： Luther-Wyrsch A,Costello E,Thali M,Buetti E,Nissen C,Surbek D,Holzgreve W,Gratwohl A,Tichelli A,Wodnar-Filipowicz A

更新日期：2001-03-01 00:00:00

abstract：:Previously, we described a nonviral cytoplasmic gene therapy vector system based on the T7 autogene concept. This system has been shown to achieve rapid and high levels of gene expression in a variety of animal cells and tissues. To test the utility of the system in vivo tumor ablation, a T7 cancer gene therapy plasmi...

journal_title：Human gene therapy

authors： Chen X,Li Y,Xiong K,Aizicovici S,Xie Y,Zhu Q,Sturtz F,Shulok J,Snodgrass R,Wagner TE,Platika D

更新日期：1998-03-20 00:00:00

abstract：:Previous studies have documented that the skin can be used as a bioreactor to produce proteins for systemic release to treat diseases. A gene-switch system has been developed that allows regulated expression of therapeutic genes. To determine whether this system could be used in the skin, we developed a transgenic mou...

journal_title：Human gene therapy

authors： Cao T,Tsai SY,O'Malley BW,Wang XJ,Roop DR

更新日期：2002-06-10 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：:Replacement of the p53 tumor suppressor gene is a rational approach to the management of malignant gliomas because p53 is frequently mutated or inactivated in these cancers. Major weaknesses of this approach are that malignant gliomas are mixtures of cells with wild-type and mutant p53, and that tumor cells exhibiting...

journal_title：Human gene therapy

authors： Ito H,Kanzawa T,Miyoshi T,Hirohata S,Kyo S,Iwamaru A,Aoki H,Kondo Y,Kondo S

更新日期：2005-06-01 00:00:00

abstract：:Current HIV-1 gene therapy approaches aim at stopping the viral life cycle at its earliest steps, such as entry or immediate postentry events. Among the most widely adopted strategies are CCR5 downregulation/knockout and the use of broadly neutralizing antibodies. However, the long-term efficacy and side effects are s...

journal_title：Human gene therapy

authors： Jung U,Urak K,Veillette M,Nepveu-Traversy MÉ,Pham QT,Hamel S,Rossi JJ,Berthoux L

更新日期：2015-10-01 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：:Adenovirus type 5 (Ad5) is a commonly used vector for gene therapy, but its efficacy is limited by high seroprevalence and off-target hepatic and splenic sequestration. In order to circumvent these limitations, the use of vectors derived from rare species adenoviruses is appealing. The opportunity to retarget rare spe...

journal_title：Human gene therapy

authors： Coughlan L,Uusi-Kerttula H,Ma J,Degg BP,Parker AL,Baker AH

更新日期：2014-04-01 00:00:00

abstract：:Gene delivery via murine-based recombinant retroviral vectors is currently widely used in gene therapy clinical trials. The vectors are engineered to be replication defective by replacing the structural and nonstructural genes of a cloned infectious retrovirus with a therapeutic gene of interest. The retroviral partic...

journal_title：Human gene therapy

authors： Martineau D,Klump WM,McCormack JE,DePolo NJ,Kamantigue E,Petrowski M,Hanlon J,Jolly DJ,Mento SJ,Sajjadi N

更新日期：1997-07-01 00:00:00

abstract：:Three dogs with deficiency of the lysosomal enzyme alpha-L-iduronidase were treated by gene replacement therapy targeted at muscle. Direct intramuscular injections of plasmid encoding the alpha-L-iduronidase gene cDNA resulted in no detectable enzyme production, but may have resulted in immunologic sensitization to id...

journal_title：Human gene therapy

authors： Shull RM,Lu X,McEntee MF,Bright RM,Pepper KA,Kohn DB

更新日期：1996-08-20 00:00:00

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：:Subcutaneous vaccination therapy with glioma cells, which are retrovirally transduced to secrete granulocyte-macrophage colony-stimulating factor (GM-CSF), has previously proven effective in C57BL/6 mice harboring intracerebral GL261 gliomas. However, clinical ex vivo gene therapy for human gliomas would be difficult,...

journal_title：Human gene therapy

authors： Herrlinger U,Jacobs A,Quinones A,Woiciechowsky C,Sena-Esteves M,Rainov NG,Fraefel C,Breakefield XO

更新日期：2000-07-01 00:00:00

abstract：:As efficient and less toxic virus-derived gene therapy vectors are developed, a pressing problem is to avoid immune response to the therapeutic gene product. Secreted therapeutic proteins potentially represent a special problem, as they are readily available to professional antigen-presenting cells throughout the body...

journal_title：Human gene therapy

authors： Cerullo V,McCormack W,Seiler M,Mane V,Cela R,Clarke C,Rodgers JR,Lee B

更新日期：2007-12-01 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：:Diabetes mellitus is associated with increased risk of heart failure. It has been previously demonstrated in mice that a single injection of adeno-associated virus 8 encoding urocortin 2 (AAV8.UCn2) increases glucose disposal in models of insulin resistance and improves the function of the failing heart. The present s...

journal_title：Human gene therapy

authors： Kim YC,Giamouridis D,Lai NC,Guo T,Xia B,Fu Z,Gao MH,Hammond HK

更新日期：2019-06-01 00:00:00

abstract：:Gene-modified lymphocytes have a potential role in the therapy of cancer, infectious diseases, and genetic disorders of the immune system. Current gene therapy protocols involving gene transfer into lymphocytes utilize retroviruses with amphotropic envelope proteins. However, transduction efficiencies in lymphocytes u...

journal_title：Human gene therapy

authors： Lam JS,Reeves ME,Cowherd R,Rosenberg SA,Hwu P

更新日期：1996-08-01 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：:Overexpression of human manganese-containing superoxide dismutase (MnSOD) activity has been demonstrated to suppress malignancy in human melanoma and breast carcinoma cells in vitro and in vivo. To study its effects on human oral squamous carcinoma cells, stable transfection and expression of MnSOD in SCC-25 cells hav...

journal_title：Human gene therapy

authors： Liu R,Oberley TD,Oberley LW

更新日期：1997-03-20 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：:Heme oxygenase 1 (HO-1) is an inducible enzyme that catalyzes heme to generate bilirubin, ferritin, and carbon monoxide. Because enhanced expression of HO-1 confers protection against many types of cell and tissue damage by modulating apoptotic cell death or cytokine expression profiles, we hypothesized that adenoviru...

journal_title：Human gene therapy

authors： Tsuburai T,Suzuki M,Nagashima Y,Suzuki S,Inoue S,Hasiba T,Ueda A,Ikehara K,Matsuse T,Ishigatsubo Y

更新日期：2002-11-01 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：:Engineered measles virus (MV) strains deriving from the vaccine lineage represent a promising oncolytic platform and are currently being tested in phase I trials. In this study, we have demonstrated that MV strains genetically engineered to express the human sodium iodide symporter (NIS) have significant antitumor act...

journal_title：Human gene therapy

authors： Opyrchal M,Allen C,Iankov I,Aderca I,Schroeder M,Sarkaria J,Galanis E

更新日期：2012-04-01 00:00:00

abstract：:The therapeutic use of neurotrophic factors for neurodegenerative diseases is promising, however, optimal methods for continuous delivery of these substances to the human central nervous system (CNS) remains problematic. One approach would be to graft genetically engineered human cells that continuously secrete high l...

journal_title：Human gene therapy

authors： Lin Q,Cunningham LA,Epstein LG,Pechan PA,Short MP,Fleet C,Bohn MC

更新日期：1997-02-10 00:00:00