Abstract:
:The optimal stem cell source for stem cell gene therapy has not been defined. Most gene transfer studies have used peripheral blood or marrow repopulating cells collected after administration of granulocyte colony-stimulating factor and stem cell factor (G-CSF/SCF). For clinical applications, however, growth factor administration may not be feasible. Thus, in the current study we used a competitive repopulation assay in the dog to directly compare transduction efficiency of steady-state marrow, G-CSF/SCF-primed marrow, and G-CSF/SCF-mobilized peripheral blood. Cells from all three sources were transduced, cryopreserved, and thawed together before infusion into myeloablated dogs. Gene marking in hematopoietic repopulating cells was assessed by polymerase chain reaction. While primed marrow resulted in the highest long-term marking levels, steady-state marrow was transduced at least as efficiently as mobilized peripheral blood in all three dogs. These results suggest that steady-state marrow may be an appropriate source for genetic modification of hematopoietic stem cells.
journal_name
Hum Gene Therjournal_title
Human gene therapyauthors
Thomasson B,Peterson L,Thompson J,Goerner M,Kiem HPdoi
10.1089/104303403322542329keywords:
subject
Has Abstractpub_date
2003-11-20 00:00:00pages
1683-6issue
17eissn
1043-0342issn
1557-7422journal_volume
14pub_type
杂志文章abstract::Glaucoma, a group of optic neuropathies, is the leading cause of irreversible blindness. Neuronal apoptosis in glaucoma is primarily associated with high intraocular pressure caused by chronically impaired outflow of aqueous humor through the trabecular meshwork, a reticulum of mitotically inactive endothelial-like ce...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/10430340152677449
更新日期:2001-11-20 00:00:00
abstract::Gene transfer for therapeutic angiogenesis represents a novel treatment for patients with chronic angina refractory to standard medical therapy and not amenable to conventional revascularization. We sought to assess the role of intraoperative multiplane transesophageal echocardiography (MPTEE) in guiding injection of ...
journal_title:Human gene therapy
pub_type: 临床试验,杂志文章
doi:10.1089/10430349950016951
更新日期:1999-09-20 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
pub_type: 杂志文章
doi:10.1089/10430349950018481
更新日期:1999-03-20 00:00:00
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journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.2019.178
更新日期:2019-08-01 00:00:00
abstract::Gene therapy for Duchenne muscular dystrophy will likely require that the corrective dystrophin gene be delivered to a high fraction of muscle fibers in vivo. Because of the large size of the dystrophin cDNA, adenoviral (Ad) vectors have been developed for this application. However, Ad vectors transduce mature muscle ...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.2006.17.314
更新日期:2006-03-01 00:00:00
abstract::The enzyme hypoxanthine-guanine phosphoribosyltransferase (HGPRT) expressed by the parasite Trypanosoma brucei (Tb) can convert allopurinol, a purine analogue, to corresponding nucleotides with greater efficiency than its human homologue. We have developed a retroviral system that expresses the parasitic enzyme and te...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/10430340152528165
更新日期:2001-09-01 00:00:00
abstract::Dystrophic epidermolysis bullosa (DEB) comprises a family of inherited blistering skin disorders for which no corrective therapy currently exists. In the most severe form, the Hallopeau-Siemens subtype (RDEB-HS), the epidermal adhesion protein collagen VII is absent from the skin as a consequence of null mutations in ...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/10430340260201743
更新日期:2002-09-01 00:00:00
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journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.2005.16.200
更新日期:2005-02-01 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
pub_type: 杂志文章
doi:10.1089/hum.2010.250
更新日期:2011-08-01 00:00:00
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journal_title:Human gene therapy
pub_type: 杂志文章,评审
doi:10.1089/104303402760128504
更新日期:2002-07-20 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
pub_type: 杂志文章
doi:10.1089/hum.2011.207
更新日期:2012-07-01 00:00:00
abstract::Recombinant adeno-associated virus 2 (rAAV) vectors have been successfully used for sustained expression of therapeutic genes. The potential of using rAAV as a cancer vaccine vector and the impact of a bacterial plasmid adjuvant on this activity were investigated. C57BL/6 mice received a single intramuscular injection...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.2004.15.856
更新日期:2004-09-01 00:00:00
abstract::Replication-deficient adenoviruses are known to induce acute injury and inflammation of infected tissues, thus limiting their use for human gene therapy. However, molecular mechanisms triggering this response have not been fully defined. To characterize this response, chemokine expression was evaluated in DBA/2 mice f...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/10430349950018364
更新日期:1999-04-10 00:00:00
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journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.2006.176
更新日期:2007-09-01 00:00:00
abstract::Hyperoxia and ischemia-reperfusion cause profound lung cellular damage mediated, in part, by generation of oxygen radicals. We hypothesized that gene therapy can be used to overcome oxidant injury by augmenting intracellular antioxidant enzymes. Adult rats were injected intratracheally with an adenovirus (Ad) vector e...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.1998.9.10-1487
更新日期:1998-07-01 00:00:00
abstract::The hepatocarcinoma-intestine-pancreas (HIP) gene, also called pancreatitis-associated protein-1 (PAP1) or Reg IIIalpha, is activated in most human hepatocellular carcinomas (HCCs) but not in normal liver, which suggests that HIP regulatory sequence could be used as efficient liver tumor-specific promoters to express ...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.2007.153
更新日期:2008-09-01 00:00:00
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journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.2014.070
更新日期:2015-01-01 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
pub_type: 临床试验,杂志文章,多中心研究,随机对照试验
doi:10.1089/104303404323142060
更新日期:2004-06-01 00:00:00
abstract::This study focuses on the design, construction, and evaluation of a chimeric promoter for gene therapy applications where it is desirable to have low-level basal expression of the newly transferred gene, which can be induced to higher levels of expression by the administration of pharmacologic agents that can be safel...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.1996.7.15-1883
更新日期:1996-10-01 00:00:00
abstract::Recombinant adenoviruses have great potential as gene delivery systems because of their ability to infect a wide range of target cells. However, systemic delivery of viral vectors to tissues other than liver and spleen has been inefficient because of the rapid clearance of the circulating virus by the liver. In the pr...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/10430340050015806
更新日期:2000-03-01 00:00:00
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journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/10430340050016166
更新日期:2000-01-01 00:00:00
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journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.2006.17.921
更新日期:2006-09-01 00:00:00
abstract::Immune cells are involved in the pathogenesis of osteoarthritis (OA). CD4(+) T cells were activated during the onset of OA and induced macrophage inflammatory protein (MIP)-1γ expression and subsequent osteoclast formation. We evaluated the effects of local knockdown of MIP-1γ in a mouse OA model induced by anterior c...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.2012.189
更新日期:2013-10-01 00:00:00
abstract::A human immunodeficiency virus type 1 (HIV-1)-based retroviral vector pseudotyped with HIV envelope containing the herpes simplex virus-thymidine kinase (HSV-TK) gene under the control of the HIV LTR promoter (pHXTKN) was constructed and stably transferred into human CD4(+) H9, CEM, and U937 cells. RNase protection as...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/10430340150218378
更新日期:2001-02-10 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
pub_type: 杂志文章
doi:10.1089/10430349950017176
更新日期:1999-09-01 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
pub_type: 杂志文章
doi:10.1089/104303404772679986
更新日期:2004-02-01 00:00:00
abstract::In summary, I will reiterate the five points I would like to leave with you today: First, the biological revolution has extraordinary power to do good. As long as the use of our new genetic knowledge is guided by the traditional ideals of the healing professions--to help improve the human condition without doing harm-...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/hum.1992.3.1-51
更新日期:1992-02-01 00:00:00
abstract::Unlike oncoretroviruses, lentiviral vectors can insert large genes and can target both dividing and nondividing cells; thus they hold unique promise as gene transfer agents. To enhance target range, the native lentiviral envelope glycoprotein is replaced (pseudotyped) with vesicular stomatitis virus G (VSVG), and the ...
journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/10430340360464723
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journal_title:Human gene therapy
pub_type: 杂志文章,评审
doi:10.1089/hum.2017.139
更新日期:2017-10-01 00:00:00
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journal_title:Human gene therapy
pub_type: 杂志文章
doi:10.1089/104303402317322276
更新日期:2002-04-10 00:00:00