Adrenomedullin gene delivery protects against cerebral ischemic injury by promoting astrocyte migration and survival.

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：:Bone marrow mesenchymal stem cells (BMSCs) represent an important source of cells for tissue repair. The tropism of these cells to the sites of injury and tumors has been well established. Their tumor-homing properties make BMSCs good candidates as antitumor agent delivery vehicles. In this study, we showed that BMSCs...

journal_title：Human gene therapy

authors： Song C,Xiang J,Tang J,Hirst DG,Zhou J,Chan KM,Li G

更新日期：2011-04-01 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：:Although recombinant adeno-associated virus serotype 8 (AAV8) and serotype 5 (AAV5) vectors have shown efficacy in Phase 1 clinical trials for gene therapy of hemophilia B, it has become increasingly clear that these serotypes are not optimal for transducing primary human hepatocytes. We have previously reported that ...

journal_title：Human gene therapy

authors： Brown HC,Doering CB,Herzog RW,Ling C,Markusic DM,Spencer HT,Srivastava A,Srivastava A

更新日期：2020-10-01 00:00:00

abstract：:Although replication-deficient adenoviruses can efficiently transfer genes to the salivary glands, the current vectors precipitate an immediate, transient decrease in salivary function. To study the cause of this salivary hypofunction, 10(6)-10(10) plaque-forming units (pfu) of the vector AdCMV beta gal were delivered...

journal_title：Human gene therapy

authors： Adesanya MR,Redman RS,Baum BJ,O'Connell BC

更新日期：1996-06-10 00:00:00

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 ad...

journal_title：Human gene therapy

authors： Thomasson B,Peterson L,Thompson J,Goerner M,Kiem HP

更新日期：2003-11-20 00:00:00

abstract：:pZIG(hGCSFR) is a retroviral vector that can co-express two genes and also provides alternative selection markers. This retroviral vector has been constructed to incorporate an internal ribosome entry site (IRES) element to co-express two exogenous genes in mammalian cells. Two marker/selection genes have been cloned ...

journal_title：Human gene therapy

authors： Saleh M

更新日期：1997-05-20 00:00:00

abstract：:Somatic gene therapy using nonautologous recombinant cells immunologically protected with alginate microcapsules has been successfully used to treat rodent genetic diseases. We now report the delivery of recombinant gene products to the brain in rodents by implanting microencapsulated cells for the purpose of eventual...

journal_title：Human gene therapy

authors： Ross CJ,Ralph M,Chang PL

更新日期：1999-01-01 00:00:00

abstract：:Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a devastating disease caused by mutations in TYMP, which encodes thymidine phosphorylase (TP). In MNGIE patients, TP dysfunction results in systemic thymidine and deoxyuridine overload, which interferes with mitochondrial DNA replication. Preclinical stu...

journal_title：Human gene therapy

authors： Cabrera-Pérez R,Vila-Julià F,Hirano M,Mingozzi F,Torres-Torronteras J,Martí R

更新日期：2019-08-01 00:00:00

abstract：:Eleven patients with moderate to severe erectile dysfunction (ED) were given a single-dose corpus cavernosum injection of hMaxi-K, a "naked" DNA plasmid carrying the human cDNA encoding hSlo (for human slow-poke), the gene for the alpha, or pore-forming, subunit of the human smooth muscle Maxi-K channel. Three patient...

journal_title：Human gene therapy

authors： Melman A,Bar-Chama N,McCullough A,Davies K,Christ G

更新日期：2006-12-01 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

abstract：:Conditionally replicative adenovirus (CRAd) vectors are designed for specific oncolytic replication in tumor tissues with concomitant sparing of normal cells. As such, CRAds offer an unprecedented level of anticancer potential for malignancies that have been refractory to previous cancer gene therapy interventions. CR...

journal_title：Human gene therapy

authors： Li H,Haviv YS,Derdeyn CA,Lam J,Coolidge C,Hunter E,Curiel DT,Blackwell JL

更新日期：2001-12-10 00:00:00

abstract：:We previously demonstrated that intramuscular plasmid injection serves as a useful method of long-term systemic delivery of cytokines. In the present study, we assess intramuscular DNA injection as a means of systemically delivering interleukin 10 (IL-10), a cytokine with immunosuppressive properties, and preventing t...

journal_title：Human gene therapy

authors： Nitta Y,Tashiro F,Tokui M,Shimada A,Takei I,Tabayashi K,Miyazaki J

更新日期：1998-08-10 00:00:00

abstract：:A major obstacle for the efficacy of cancer gene therapy is the need to transduce a high proportion of tumor cells with genes that directly or indirectly cause their death. During the formation of certain organs, cells compete among themselves to colonize the whole tissue. We reasoned that cell competition could be us...

journal_title：Human gene therapy

authors： Martinez-Quintanilla J,Cascallo M,Fillat C,Alemany R

更新日期：2009-07-01 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：:Human serum is known to inactivate many retroviruses, including murine leukemia viruses (MLV). Exposure of vectors based on MLV to human serum components would presumably decrease the efficiency of gene transfer in vivo. Human serum also lyses xenogeneic cells, which would affect the survival of retroviral vector pack...

journal_title：Human gene therapy

authors： Russell DW,Berger MS,Miller AD

更新日期：1995-05-01 00:00:00

abstract：:The potential of short interfering RNA (siRNA) to be developed for therapeutic use against cancer depends on the availability of an efficient tumor-specific delivery vehicle. We have previously shown that a nanoscale nonviral liposome-based complex that includes an anti-transferrin receptor single-chain antibody fragm...

journal_title：Human gene therapy

authors： Pirollo KF,Zon G,Rait A,Zhou Q,Yu W,Hogrefe R,Chang EH

更新日期：2006-01-01 00:00:00

abstract：:Establishing pharmacological parameters, such as efficacy, routes of administration, and toxicity, for recombinant adeno-associated virus (rAAV) vectors is a prerequisite for gaining acceptance for clinical applications. In fact, even a therapeutic window, that is, the dose range between therapeutic efficacy and toxic...

journal_title：Human gene therapy

authors： Virag T,Cecchini S,Kotin RM

更新日期：2009-08-01 00:00:00

abstract：:Gene therapy studies in primates can provide important information regarding vector tropism, specific cellular expression, biodistribution, and safety prior to clinical trials. In this study, we report the assessment of transduction efficiency of recombinant adeno-associated virus (rAAV) vectors using human postmortem...

journal_title：Human gene therapy

authors： Fradot M,Busskamp V,Forster V,Cronin T,Léveillard T,Bennett J,Sahel JA,Roska B,Picaud S

更新日期：2011-05-01 00:00:00

abstract：:Despite efforts toward improvements in retrovirus-mediated gene transfer, stable high-level expression of a therapeutic gene in human hematopoietic stem cells remains a great challenge. We have evaluated the efficiency of different viral long terminal repeats (LTRs) in long-term expression of a transgene in vivo, usin...

journal_title：Human gene therapy

authors： Kaneko S,Onodera M,Fujiki Y,Nagasawa T,Nakauchi H

更新日期：2001-01-01 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：:The inherited deficiency in adenosine deaminase (ADA), which results in severe combined immunodeficiency, is generally regarded as an optimal model for the development of human somatic gene therapy. The ideal target for the correction of ADA deficiency and other lympho-hematopoietic disorders would be the hematopoieti...

journal_title：Human gene therapy

authors： Cournoyer D,Scarpa M,Mitani K,Moore KA,Markowitz D,Bank A,Belmont JW,Caskey CT

更新日期：1991-10-01 00:00:00

abstract：:We have shown that adenovirus-mediated manipulation of apoptotic genes such as bax could be a therapeutic option for prostate cancer. Unfortunately, the response of experimental prostate tumors to a single therapeutic gene of the apoptotic pathway is short-lived, and most of these tumors relapse after a short period o...

journal_title：Human gene therapy

authors： Zhang Y,Yu J,Unni E,Shao TC,Nan B,Snabboon T,Kasper S,Andriani F,Denner L,Marcelli M

更新日期：2002-11-20 00:00:00

abstract：:Cell-based therapy for muscular dystrophies was initiated in humans after promising results obtained in murine models. Early trials failed to show substantial clinical benefit, sending researchers back to the bench, which led to the discovery of many hurdles as well as many new venues to optimize this therapeutic stra...

journal_title：Human gene therapy

authors： Negroni E,Bigot A,Butler-Browne GS,Trollet C,Mouly V

更新日期：2016-02-01 00:00:00

abstract：:Traditionally, skeletal muscle and liver are the preferred target organs for gene transfer to supply a transgene product into the systemic circulation. In this respect, adipose tissue presents a number of attractive features. However, adipose tissue transduction in vivo has not been feasible by conventional methods. T...

journal_title：Human gene therapy

authors： Mizukami H,Mimuro J,Ogura T,Okada T,Urabe M,Kume A,Sakata Y,Ozawa K

更新日期：2006-09-01 00:00:00

abstract：:The efficient and specific introduction of genes into cancer cells in vivo remains a major challenge for current gene therapy modalities. Peptides possess appropriate properties to serve as tumor-targeting agents. Thus, finding new cancer-selective peptides directing gene transfer to neoplastic cells by reducing trans...

journal_title：Human gene therapy

authors： Böckmann M,Hilken G,Schmidt A,Cranston AN,Tannapfel A,Drosten M,Frilling A,Ponder BA,Pützer BM

更新日期：2005-11-01 00:00:00

abstract：:Guidelines for testing gene therapy products for ecotropic replication-competent retrovirus (Eco-RCR) have not been delineated as they have for amphotropic viruses. To evaluate biologic assays that can detect these viruses, we compared an S(+)/L(-) assay and a marker rescue assay designed specifically for Eco-RCR dete...

journal_title：Human gene therapy

authors： Reeves L,Duffy L,Koop S,Fyffe J,Cornetta K

更新日期：2002-09-20 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

authors： Rajagopalan S,Olin JW,Young S,Erikson M,Grossman PM,Mendelsohn FO,Regensteiner JG,Hiatt WR,Annex BH

更新日期：2004-06-01 00:00:00

abstract：:Transfer of a herpes simplex virus-derived thymidine kinase (HSV-tk) gene into brain tumor cells and subsequent ganciclovir (GCV) treatment has been shown by others to be an effective treatment in rats with intracerebrally inoculated 9L gliosarcomas. Mechanism of action and reproducibility are, however, still a matter...

journal_title：Human gene therapy

authors： Vincent AJ,Vogels R,Someren GV,Esandi MC,Noteboom JL,Avezaat CJ,Vecht C,Bekkum DW,Valerio D,Bout A,Hoogerbrugge PM

更新日期：1996-01-20 00:00:00

abstract：:Adenovirus-polylysine-DNA complexes were evaluated for their capacity to accomplish direct in vivo gene transfer to airway epithelium employing a rodent model. Binary complexes containing transferrin or adenovirus, or combination complexes containing both transferrin and adenovirus, were evaluated. The highest in vitr...

journal_title：Human gene therapy

authors： Gao L,Wagner E,Cotten M,Agarwal S,Harris C,Rømer M,Miller L,Hu PC,Curiel D

更新日期：1993-02-01 00:00:00