Efficiency of high- and low-voltage pulse combinations for gene electrotransfer in muscle, liver, tumor, and skin.

abstract：:Immunoisolation of allogeneic cells within a membrane-bound device is a unique approach for gene therapy. We employed an immunoisolation device that protects allograft, but not xenograft, cells from destruction, to implant a human fibroblast line (MSU 1.2) in athymic rodents. Cells, transduced with the MFG-human facto...

journal_title：Human gene therapy

authors： Brauker J,Frost GH,Dwarki V,Nijjar T,Chin R,Carr-Brendel V,Jasunas C,Hodgett D,Stone W,Cohen LK,Johnson RC

更新日期：1998-04-10 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：: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

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：:Replication-deficient adenovirus vector (Ad) is one of the most efficient gene transfer vehicles for human gene therapy. However, Ad is antigenic, known to evoke prominent inflammatory responses in vivo, and there are concerns that using Ad in patients with immune-mediated disorders (allergy and autoimmune diseases) m...

journal_title：Human gene therapy

authors： Suzuki M,Suzuki S,Yamamoto N,Komatsu S,Inoue S,Hashiba T,Nishikawa M,Ishigatsubo Y

更新日期：2000-04-10 00:00:00

abstract：:In the next decades, gene editing technologies are expected to be used in the treatment and prevention of human diseases. Yet, the future uses of gene editing in medicine are still unknown, including its applicability and effectiveness to the treatment and prevention of infectious diseases, cancer, and monogenic and p...

journal_title：Human gene therapy

authors： Rocha LFM,Braga LAM,Mota FB

更新日期：2020-08-01 00:00:00

abstract：:As an alternative to virus-mediated gene transfer, we previously demonstrated a simple, safe, and efficient transfer of foreign gene into the central nervous system using continuous injection of a plasmid DNA-cationic liposome complex. To explore whether this approach can be applied to the treatment of certain neurolo...

journal_title：Human gene therapy

authors： Imaoka T,Date I,Ohmoto T,Nagatsu T

更新日期：1998-05-01 00:00:00

abstract：:Introduction of a new vaccine requires choosing a delivery system that provides safe administration and the desired level of immunogenicity. The safety, tolerability, and immunogenicity of three monthly 2.5-mg doses of a PfCSP DNA vaccine were evaluated in healthy volunteers as administered intramuscularly (IM) by nee...

journal_title：Human gene therapy

authors： Epstein JE,Gorak EJ,Charoenvit Y,Wang R,Freydberg N,Osinowo O,Richie TL,Stoltz EL,Trespalacios F,Nerges J,Ng J,Fallarme-Majam V,Abot E,Goh L,Parker S,Kumar S,Hedstrom RC,Norman J,Stout R,Hoffman SL

更新日期：2002-09-01 00:00:00

abstract：:The management of disorders of the nervous system remains a medical challenge. The key goals are to understand disease mechanisms, to validate therapeutic targets, and to develop new therapeutic strategies. Viral vector-mediated gene transfer can meet these goals and vectors based on lentiviruses have particularly use...

journal_title：Human gene therapy

authors： Wong LF,Goodhead L,Prat C,Mitrophanous KA,Kingsman SM,Mazarakis ND

更新日期：2006-01-01 00:00:00

abstract：:Naked plasmid DNA electrotransfer offers advantages over viral-based gene delivery, including being regulatory permissive, but factors influencing expression efficiency and cell fate impact on translational utility. This study compared co-expression of red and green fluorescence reporter plasmids with differing promot...

journal_title：Human gene therapy

authors： Pinyon JL,Klugmann M,Lovell NH,Housley GD

更新日期：2019-02-01 00:00:00

abstract：:We reported that SNV-derived retroviral vectors, which display single-chain antibodies on the viral surface, enable cell type-specific gene delivery into various human cells. In particular, the SNV cell type-specific gene delivery vector system appears to be well suited to transduce genes into cells of the human hemat...

journal_title：Human gene therapy

authors： Jiang A,Fisher H,Pomerantz RJ,Dornburg R

更新日期：1999-11-01 00:00:00

abstract：:Mutations in the alpha-chain of lysosomal hexosaminidase (EC 3.2.1.52) underlie two distinct biochemical phenotypes known as variant B and variant B1 of G(M2) gangliosidosis. This paper shows that the transduction of human B1-type fibroblasts (producing catalytically inactive alpha-chains) with a retroviral vector enc...

journal_title：Human gene therapy

authors： Teixeira CA,Sena-Esteves M,Lopes L,Sá Miranda MC,Ribeiro MG

更新日期：2001-09-20 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：:Adrenomedullin (AM) has been shown to protect against ischemia/reperfusion-induced myocardial infarction and apoptosis. In the present study, we examined the potential neuroprotective action of delayed AM gene transfer in cerebral ischemia. Three days after a 1-hr occlusion of the middle cerebral artery (MCAO), rats w...

journal_title：Human gene therapy

authors： Xia CF,Yin H,Borlongan CV,Chao J,Chao L

更新日期：2004-12-01 00:00:00

abstract：:To target expression of toxic genes to Epstein-Barr virus (EBV)-associated tumor cells, we have developed an EBV-driven enzyme prodrug system (EDEPS) that takes advantage of the trans-activating properties of EBNA1, a latent protein expressed in all EBV-containing cells, to direct expression of cytosine deaminase (CD)...

journal_title：Human gene therapy

authors： Judde JG,Spangler G,Magrath I,Bhatia K

更新日期：1996-03-20 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：:Efficient DNA electrotransfer can be achieved with combinations of short high-voltage (HV) and long low voltage (LV) pulses that cover two effects of the pulses, namely, target cell electropermeabilization and DNA electrophoresis within the tissue. Because HV and LV can be delivered with a lag up to 3000 sec between t...

journal_title：Human gene therapy

authors： Satkauskas S,André F,Bureau MF,Scherman D,Miklavcic D,Mir LM

更新日期：2005-10-01 00:00:00

abstract：:Niemann-Pick type C (NP-C) disease is a neurodegenerative disorder characterized neuropathologically by ballooned neurons distended with lipid storage and widespread neuronal loss. Neural stem cells (NSC) derived from NP-C disease models have decreased ability for self-renewal and neuronal differentiation. Investigati...

journal_title：Human gene therapy

authors： Lee H,Kang JE,Lee JK,Bae JS,Jin HK

更新日期：2013-07-01 00:00:00

abstract：:Three-vessel disease (TVD) is a severe coronary heart disease (CHD) with poor prognosis. Niemann-Pick C1-like 1 (NPC1L1) is a transporter protein for exogenous cholesterol absorption, and 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) is a rate-limiting enzyme for cholesterol synthesis. We aimed to investigat...

journal_title：Human gene therapy

authors： Zhao X,Li J,Tang X,Liu R,Xu J,Xu L,Jiang L,Huang K,Tian J,Feng X,Wu Y,Zhang Y,Wang D,Sun K,Xu B,Zhao W,Hui R,Gao R,Song L,Yuan J

更新日期：2021-01-22 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：: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：:Chronic inflammation in tibialis anterior muscles of mdx mice was produced by a single injection of a recombinant adenovirus vector (AV) expressing an immunogenic beta-galactosidase (beta-gal). In regions of intense beta-gal staining, mononuclear infiltrates abounded, and muscle fibers showed strong extrasynaptic utro...

journal_title：Human gene therapy

authors： Waheed I,Gilbert R,Nalbantoglu J,Guibinga GH,Petrof BJ,Karpati G

更新日期：2005-04-01 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：:Antitumor gene therapy using herpes simplex type 1 thymidine kinase (TKh) and ganciclovir (GCV) treatment has revealed an important intratumoral bystander effect. A whole tumor can be eliminated when only a fraction of its tumor cells express TKh. We now report that the bystander effect not only acts within a tumor, b...

journal_title：Human gene therapy

authors： Kianmanesh AR,Perrin H,Panis Y,Fabre M,Nagy HJ,Houssin D,Klatzmann D

更新日期：1997-10-10 00:00:00

abstract：:Therapeutic levels of expression of the beta-globin gene have been difficult to achieve with conventional retroviral vectors without the inclusion of DNase I-hypersensitive site (HS2, HS3, and HS4) enhancer elements. We generated recombinant adeno-associated viral (AAV) vectors carrying an antisickling human beta-glob...

journal_title：Human gene therapy

authors： Maina N,Zhong L,Li X,Zhao W,Han Z,Bischof D,Aslanidi G,Zolotukhin S,Weigel-Van Aken KA,Rivers AE,Slayton WB,Yoder MC,Srivastava A

更新日期：2008-04-01 00:00:00

abstract：:Adult T cell leukemia/lymphoma (ATL) is derived from CD4+ T cells and has a poor prognosis because of its resistance to chemotherapy. To evaluate the effectiveness of gene therapy for ATL, the effect of ganciclovir on ATL cell lines transfected with the thymidine kinase gene of herpes simplex virus type 1 (HSV-TK) was...

journal_title：Human gene therapy

authors： Obaru K,Fujii S,Matsushita S,Shimada T,Takatsuki K

更新日期：1996-12-01 00:00:00

abstract：:Recombinant adeno-associated virus serotype 2 (rAAV2)-based human gene therapy for cystic fibrosis has progressed through a series of preclinical studies and phase I and II clinical trials. This agent has shown an encouraging safety profile, consistent levels of DNA transfer, and positive evidence of short-term clinic...

journal_title：Human gene therapy

authors： Flotte TR,Schwiebert EM,Zeitlin PL,Carter BJ,Guggino WB

更新日期：2005-08-01 00:00:00

abstract：:Recombinant poxviruses expressing immunomodulatory molecules together with specific antigens represent powerful vaccines for cancer immunotherapy. Recently, we and others have demonstrated, in vitro and in vivo, that coexpression of CD80 and CD86 costimulatory molecules enhances the immunogenic capacity of a recombina...

journal_title：Human gene therapy

authors： Feder-Mengus C,Schultz-Thater E,Oertli D,Marti WR,Heberer M,Spagnoli GC,Zajac P

更新日期：2005-03-01 00:00:00

abstract：:The ability to control proliferation of grafted cells in the heart and consequent graft size could dramatically improve the efficacy of cell therapies for cardiac repair. To achieve targeted graft cell proliferation, we created a chimeric receptor (F36Vfgfr-1) composed of a modified FK506-binding protein (F36V) fused ...

journal_title：Human gene therapy

authors： Stevens KR,Rolle MW,Minami E,Ueno S,Nourse MB,Virag JI,Reinecke H,Murry CE

更新日期：2007-05-01 00:00:00

abstract：:The first report of in vivo gene delivery to the retina dates back to 1987 when a retroviral vector was injected intraocularly in newborn mice. Later came the observation that retinal cells could be successfully transduced using adenoviral and then adeno-associated and lentiviral vectors. By 2000, it had become clear ...

journal_title：Human gene therapy

authors： Auricchio A,Smith AJ,Ali RR

更新日期：2017-11-01 00:00:00