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In Vivo Gene Transfer Strategies to Achieve

by Jonathan Rosenberg, See Profile, Stefano Rivella, Stephen M. Kaminsky, Ronald G. Crystal , 2016
"... All in-text references underlined in blue are linked to publications on ResearchGate, letting you access and read them immediately. ..."
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All in-text references underlined in blue are linked to publications on ResearchGate, letting you access and read them immediately.

Commentary Ex vivo gene transfer in the years to come

by Thomas Pap, Renate E Gay, Ulf Müller-ladner, Steffen Gay , 2001
"... Synovial fibroblasts (SFs) have become a major target for ex vivo gene transfer in rheumatoid arthritis (RA), but efficient transduction of RA-SFs still is a major problem. The low proliferation rate and heterogeneity of RA-SFs, together with their lack of highly specific surface receptors, have ham ..."
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Synovial fibroblasts (SFs) have become a major target for ex vivo gene transfer in rheumatoid arthritis (RA), but efficient transduction of RA-SFs still is a major problem. The low proliferation rate and heterogeneity of RA-SFs, together with their lack of highly specific surface receptors, have

Tolerance induction by viral in vivo gene transfer

by Eric Dobrzynski, W. Herzog - Clin. Med. Res , 2005
"... Treatment of genetic disease by protein or gene replacement therapy is hampered by immune responses to the therapeutic protein.An excellent example is formation of inhibitory antibodies to coagulation factors in treatment of the X-linked bleeding disorder hemophilia. Experiments in murine and canine ..."
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and clonal deletion. Taken together, these data indicate the potential for viral in vivo gene transfer not only to provide sustained systemic expression, but moreover to induce immunological hypo-responsiveness to the therapeutic gene product.

vivo gene transfer using cetylated polyethylenimine

by Er Sochanik, Tomasz Cichoń, Monika Makselon, Małgorzata Stróżyk, Ryszard Smolarczyk, Joanna Jazowiecka-rakus, Stanisław Szala - Acta Biochim Pol
"... This report describes gene transfer in vitro as well as in vivo using cetylated low-molecular mass (600 Da) polyethylenimine (28 % of amine groups substituted with cetyl moieties), termed CT-PEI. This compound is hydrophobic and has to be in-corporated into liposomes in order to be suitable for gene ..."
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This report describes gene transfer in vitro as well as in vivo using cetylated low-molecular mass (600 Da) polyethylenimine (28 % of amine groups substituted with cetyl moieties), termed CT-PEI. This compound is hydrophobic and has to be in-corporated into liposomes in order to be suitable

In Vivo Gene Transfer Into Murine Corneal Endothelial

by Trabecular Meshwork Cells, Donald L. Budenz, Jean Bennett, Laura Alonso, Albert Maguire
"... Purpose. To determine whether a reporter gene can be introduced into adult mammalian corneal endothelial and trabecular meshwork cells in vivo using a recombinant replicationdeficient adenovirus. Methods. Purified replication-deficient adenovirus containing the cytomegalovirus-promoted Escherichia c ..."
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expressed lacZ as measured by /3-Gal assay. Conclusions. A functional gene can be transferred in vivo into adult mammalian corneal endothelial and trabecular meshwork cells using a replication-defective adenoviral vector. Gene expression is relatively short-lived compared to that demonstrated previously

In Vivo Gene Transfer, Koch's Postulates, and Renal Disease Editorial

by unknown authors
"... In this issue of The Journal, Isaka et al. ( 1) report on the suc-cessful transfection of the human PDGF B chain gene and the TGF-f gene into the rat kidney (1). A liposomal delivery sys-tem was prepared using hemagglutinating virus of Japan to facilitate fusion of the liposome to cell membranes, an ..."
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; in contrast, TGF-fl-transfected glomeruli primarily demonstrated mesan-gial matrix expansion characteristic of a sclerotic process ( 1). In vivo gene transfer represents a major research break-through from the perspective of understanding disease patho-genesis, as well as for potential gene therapy

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

by Luigi Naldini, Ulrike Blomer, Daniel Ory, Richard Mulligan, Fred H. Gage, Inder M. Verma, Didier Trono - Science , 1996
"... A retroviral vector system based on the human immunodeficiency virus (HIV) was de-veloped that, in contrast to a murine leukemia virus-based counterpart, transduced heterologous sequences into HeLa cells and rat fibroblasts blocked in the cell cycle, as well as into human primary macrophages. Additi ..."
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. Additionally, the HIV vector could mediate stable in vivo gene transfer into terminally differentiated neurons. The ability of HIV-based viral vectors to deliver genes in vivo into nondividing cells could increase the applicability of retroviral vectors in human gene therapy. Until now, gene therapy protocols

with recombinant plasmid

by Min-fu Tsan, Julie E. White, Barbara Shepard, Julie E. White, Barbara Shepard
"... direct in vivo gene transfer ..."
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direct in vivo gene transfer

Rapid Communication In Vivo Gene Transfer and Expression in Normal Uninjured Blood

by Vessels Using, Replication-deficient Recombinant, Patricia Lemarch, Michael Jones, Izumi Yamada, Ronald G. Crystal , 1132
"... Replication-deficient recombinant adenovirus vectors do not require target cell replication for transfer and expression of exogenous genes and thus may be useful for in vivo gene therapy in the endothelium. To evaluate the feasibility of adenovirus-mediated gene transfer in vivo in normal intact blo ..."
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Replication-deficient recombinant adenovirus vectors do not require target cell replication for transfer and expression of exogenous genes and thus may be useful for in vivo gene therapy in the endothelium. To evaluate the feasibility of adenovirus-mediated gene transfer in vivo in normal intact

Ex vivo gene transfer for improved adoptive immunotherapy of cancer

by Minhtran C. Ngo, Cliona M. Rooney, Jeffrey M. Howard, Helen E. Heslop , 2011
"... Adoptive immunotherapy is an appealing approach to cancer treatment, with the potential for more precise targeting and reduced toxicity. While early clinical trial data using adoptive T cells against post-transplant virus-associated hematologic malignancies, lymphoma and melanoma have been promising ..."
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cell antigen specificity. Gene transfer may also allow expression of homeostatic cytokines or their receptors to overcome the lack of stimulatory signals or expression of dominant-negative receptors for inhibitory cytokines to compensate for an immuno-suppressive tumor milieu. In addition, suicide
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