Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.

abstract：:Gene therapy has evolved into a tempting strategy for the management of cancer and other life-threatening diseases. Various approaches employ retroviral vectors to deliver the therapeutic gene. The profound knowledge about retrovirus biology allows the generation of increasingly advanced vector systems as well as an a...

journal_title：Human gene therapy

authors： Brandtner EM,Kodajova P,Knapp E,Ertl R,Tabotta W,Salmons B,Günzburg WH,Hohenadl C

更新日期：2008-01-01 00:00:00

abstract：:Severe fetal growth restriction (FGR) affects 1:500 pregnancies, is untreatable and causes serious neonatal morbidity and death. Reduced uterine blood flow (UBF) and lack of bioavailable VEGF due to placental insufficiency is a major cause. Transduction of uterine arteries in normal or FGR sheep and guinea pigs using ...

journal_title：Human gene therapy

authors： Rossi C,Lees M,Mehta V,Heikura T,Martin J,Zachary I,Spencer R,Peebles DM,Shaw R,Karhinen M,Yla-Herttuala S,David AL

更新日期：2020-11-01 00:00:00

abstract：:Gyrate atrophy is a progressive blindness associated with deficiency of ornithine aminotransferase (OAT). The strategy of using an autologous keratinocyte graft, modified to express high levels of OAT as an ornithine-catabolizing skin-based enzyme sink, is investigated. Two OAT-containing retroviral vectors were const...

journal_title：Human gene therapy

authors： Jensen TG,Sullivan DM,Morgan RA,Taichman LB,Nussenblatt RB,Blaese RM,Csaky KG

更新日期：1997-11-20 00:00:00

abstract：:With the aim of developing new gene transfer tools for treating CF with gene therapy, we have synthesized a novel family of molecules named cationic phosphonolipids. The most efficient among them were selected by in vitro screening to compare their activities in vivo in mouse lungs. We used a reporter gene whose activ...

journal_title：Human gene therapy

authors： Guillaume-Gable C,Floch V,Mercier B,Audrézet MP,Gobin E,Le Bolch G,Yaouanc JJ,Clément JC,des Abbayes H,Leroy JP,Morin V,Férec C

更新日期：1998-11-01 00:00:00

abstract：:Following in vivo recombinant adeno-associated virus (rAAV)-based gene transfer, adaptive immune responses specific to the vector or the transgene product have emerged as a potential roadblock to successful clinical translation. The occurrence of such responses depends on several parameters, including the route of vec...

journal_title：Human gene therapy

authors： Gernoux G,Guilbaud M,Dubreil L,Larcher T,Babarit C,Ledevin M,Jaulin N,Planel P,Moullier P,Adjali O

更新日期：2015-01-01 00:00:00

abstract：:Current clinical gene therapy protocols for the treatment of human immunodeficiency virus type 1 (HIV-1) infection often involve the ex vivo transduction and expansion of CD4+ T cells derived from HIV-positive patients at a late stage in their disease (CD4 count <400). These protocols involve the transduction of T cel...

journal_title：Human gene therapy

authors： Poznansky MC,Foxall R,Mhashilkar A,Coker R,Jones S,Ramstedt U,Marasco W

更新日期：1998-03-01 00:00:00

abstract：:Myelosuppression is the main side effect of cancer chemotherapy. An improved rate of retroviral vector-mediated gene transfer to hematopoietic stem cells, shown in more recent clinical trials, has created the basis to test the concept of myeloprotective gene therapy. We transplanted clinical-scale human peripheral blo...

journal_title：Human gene therapy

authors： Schiedlmeier B,Schilz AJ,Kühlcke K,Laufs S,Baum C,Zeller WJ,Eckert HG,Fruehauf S

更新日期：2002-01-20 00:00:00

abstract：:Human papillomavirus type 16 (HPV16) is associated with the development of anogenital cancers and their precursor lesions, intraepithelial neoplasia. Treatment strategies against HPV-induced intraepithelial neoplasia are not HPV specific and mostly consist of physical removal or ablation of lesions. We had previously ...

journal_title：Human gene therapy

authors： Sharma R,Palefsky JM

更新日期：2010-07-01 00:00:00

abstract：:Reporter gene-based molecular imaging can provide invaluable information on the fate of cellular therapies postimplantation. Integrating lentiviral vectors (ILVs) are commonly used for stably engineering cells; however, their potential for insertional mutagenesis poses a significant safety concern and barrier to wides...

journal_title：Human gene therapy

authors： Hamilton AM,Foster PJ,Ronald JA

更新日期：2018-10-01 00:00:00

abstract：:Baculovirus vectors recently have been shown to be capable of efficient transduction of human hepatoma cells and primary hepatocytes in culture. This paper describes the generation of a novel recombinant baculovirus (VGZ3) in which the vesicular stomatitis virus glycoprotein G (VSV G) is present in the viral envelope....

journal_title：Human gene therapy

authors： Barsoum J,Brown R,McKee M,Boyce FM

更新日期：1997-11-20 00:00:00

abstract：:Gene therapy for heart diseases requires availability of an efficient vector for gene transfer into myocardium. Recombinant adenovirus expressing the Escherichia coli beta-galactosidase (beta-Gal) gene was shown to infect rat cardiocytes efficiently in vivo. However, a time course of gene expression showed that transg...

journal_title：Human gene therapy

authors： Gilgenkrantz H,Duboc D,Juillard V,Couton D,Pavirani A,Guillet JG,Briand P,Kahn A

更新日期：1995-10-01 00:00:00

abstract：:Clinical gene transfer research has involved adult and child subjects, and it is expected that gene transfer in fetal subjects will occur in the future. Some genetic diseases have serious adverse effects on the fetus before birth, and there is hope that prenatal gene therapy could prevent such disease progression. Res...

journal_title：Human gene therapy

authors： Strong C

更新日期：2011-11-01 00:00:00

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

authors： Loewen N,Fautsch MP,Peretz M,Bahler CK,Cameron JD,Johnson DH,Poeschla EM

更新日期：2001-11-20 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：:Rat myoblast primary cultures were tested as a model for proinsulin synthesis and processing and unregulated insulin delivery for insulin-dependent diabetes mellitus (IDDM) gene therapy. Three human proinsulin cDNA constructs containing genetically engineered furin endoprotease cleavage sites between the B-chain and C...

journal_title：Human gene therapy

authors： Simonson GD,Groskreutz DJ,Gorman CM,MacDonald MJ

更新日期：1996-01-01 00:00:00

abstract：:Successful gene transfer into articular cartilage is a prerequisite for gene therapy of articular joint disorders. In the present study we tested the hypothesis that recombinant adeno-associated virus (rAAV) vectors are capable of effecting gene transfer in isolated articular chondrocytes in vitro, articular cartilage...

journal_title：Human gene therapy

authors： Madry H,Cucchiarini M,Terwilliger EF,Trippel SB

更新日期：2003-03-01 00:00:00

abstract：:To achieve effective gene therapy, it is necessary to selectively and efficiently transfect therapeutic gene into targeted cells. In this study, we developed a combination method using mannosylated lipoplexes, which show selectivity to antigen-presenting cells such as macrophages and dendritic cells, and bubble liposo...

journal_title：Human gene therapy

authors： Un K,Kawakami S,Suzuki R,Maruyama K,Yamashita F,Hashida M

更新日期：2010-01-01 00:00:00

abstract：:Deoxyribozymes, or DNA enzymes (DNAzymes), are novel nucleic acids that have the ability to bind to specific sequences of RNA, and to cleave the target site catalytically. DNAzymes are smaller and more efficient enzymatically than ribozymes (RZs), which are catalytic nucleic acids synthesized from ribonucleotides. We ...

journal_title：Human gene therapy

authors： Wu Y,Yu L,McMahon R,Rossi JJ,Forman SJ,Snyder DS

更新日期：1999-11-20 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：:Activation of T cells is necessary for efficient retroviral-mediated gene transfer. In addition, if the population of infused cells is to be limited to transduced cells, a means of positive selection is required. We describe a clinical scale procedure for activation of donor T cells with anti-CD3/CD28 beads followed b...

journal_title：Human gene therapy

authors： Orchard PJ,Blazar BR,Burger S,Levine B,Basso L,Nelson DM,Gordon K,McIvor RS,Wagner JE,Miller JS

更新日期：2002-05-20 00:00:00

abstract：:Defined serum-free conditions have great conceptual advantages for the biological safety and standardization of clinical gene transfer into hematopoietic stem cells. In the only study reported to date, Sekhar et al. achieved low serum conditions by a complex concentration procedure of a retroviral supernatant initiall...

journal_title：Human gene therapy

authors： Glimm H,Flügge K,Möbest D,Hofmann VM,Postmus J,Henschler R,Lange W,Finke J,Kiem HP,Schulz G,Rosenthal F,Mertelsmann R,von Kalle C

更新日期：1998-04-10 00:00:00

abstract：:Vectors based on lentiviruses have become potent tools for efficient gene transfer to multiple cell types both in vitro and in vivo. In part this is attributable to the stability of transduction afforded by integration into the target cell genome. However, evidence indicates that episomal forms of the vector can also ...

journal_title：Human gene therapy

authors： Philpott NJ,Thrasher AJ

更新日期：2007-06-01 00:00:00

abstract：:Doxycycline (DOX) is widely used as a pharmacological agent and as an effector molecule in inducible gene expression systems. For most applications, it is important to determine whether the DOX concentration reaches the level required for optimal efficacy. We developed a sensitive bioassay for measuring the DOX concen...

journal_title：Human gene therapy

authors： Kleibeuker W,Zhou X,Centlivre M,Legrand N,Page M,Almond N,Berkhout B,Das AT

更新日期：2009-05-01 00:00:00

abstract：:Here we show potent inhibition of HIV-1 replication in a human T cell line and primary human CD4(+) cells by expressing a single antiviral protein. Nullbasic is a mutant form of the HIV-1 Tat protein that was previously shown to strongly inhibit HIV-1 replication in nonhematopoietic cell lines by targeting three steps...

journal_title：Human gene therapy

authors： Apolloni A,Lin MH,Sivakumaran H,Li D,Kershaw MH,Harrich D

更新日期：2013-03-01 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：:Efficient and homogeneous gene transfer to cardiac myocytes is a major target in myocardial gene therapy. The aim of this study was to determine the conditions permitting efficient, homogeneous, adenovirus-mediated gene transfer to cardiac myocytes, with a view to application during coronary artery catheterization. Ge...

journal_title：Human gene therapy

authors： Logeart D,Hatem SN,Rücker-Martin C,Chossat N,Névo N,Haddada H,Heimburger M,Perricaudet M,Mercadier JJ

更新日期：2000-05-01 00:00:00

abstract：:Retinal gene therapy based on adeno-associated viral (AAV) vectors is safe and efficient in humans. The low intrinsic DNA transfer capacity of AAV has been expanded by dual vectors where a large expression cassette is split in two halves independently packaged in two AAV vectors. Dual AAV transduction efficiency, howe...

journal_title：Human gene therapy

authors： Maddalena A,Dell'Aquila F,Giovannelli P,Tiberi P,Wanderlingh LG,Montefusco S,Tornabene P,Iodice C,Visconte F,Carissimo A,Medina DL,Castoria G,Auricchio A

更新日期：2018-08-01 00:00:00

abstract：:We studied hematopoietic progenitors from fetal baboon blood, marrow, and liver at four time points (125, 140, 160, and 175 days) during the third trimester (gestation approximately 180 days) to determine if fetal baboons might be an appropriate model for in utero gene therapy of hematopoietic stem cells (HSCs). Cells...

journal_title：Human gene therapy

authors： Winkler A,Kiem HP,Shields LE,Sun QH,Andrews RG

更新日期：1999-03-01 00:00:00

abstract：:Due to both the avascularity of the cornea and the relatively immune-privileged status of the eye, corneal transplantation is one of the most successful clinical transplant procedures. However, in high-risk patients, which account for >20% of the 180,000 transplants carried out worldwide each year, the rejection rate ...

journal_title：Human gene therapy

authors： Fouladi N,Parker M,Kennedy V,Binley K,McCloskey L,Loader J,Kelleher M,Mitrophanous KA,Stout JT,Ellis S

更新日期：2018-06-01 00:00:00

abstract：:The immune response against human immunodeficiency virus type-1 (HIV-1) is believed to play a role in controlling the early stages of disease progression. The cellular immune response, in particular cytotoxic T lymphocyte (CTL) activity, may be important for eliminating virally infected cells in HIV-1-infected individ...

journal_title：Human gene therapy

authors： Laube LS,Burrascano M,Dejesus CE,Howard BD,Johnson MA,Lee WT,Lynn AE,Peters G,Ronlov GS,Townsend KS

更新日期：1994-07-01 00:00:00