Capsid-modified adenoviral vectors for improved muscle-directed gene therapy.

abstract：:Recombinant adenoviruses have received much attention as a potential vector for gene therapy because of their ability to transduce many cell types with high efficiencies in vivo. After intravenous infusion, the majority of the vector is found in hepatocytes, but vector DNA is found to varying degrees in other tissues....

journal_title：Human gene therapy

authors： Peeters MJ,Patijn GA,Lieber A,Meuse L,Kay MA

更新日期：1996-09-10 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：:Recent marketing approval for genetically engineered hematopoietic stem and T cells bears witness to the substantial improvements in lentiviral vectors over the last two decades, but evaluations of the long-term efficacy and toxicity of gene and cell therapy products will, nevertheless, require further studies in nonh...

journal_title：Human gene therapy

authors： Sii-Felice K,Castillo Padilla J,Relouzat F,Cheuzeville J,Tantawet S,Maouche L,Le Grand R,Leboulch P,Payen E

更新日期：2019-10-01 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

authors： Muruve DA,Barnes MJ,Stillman IE,Libermann TA

更新日期：1999-04-10 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：:Previously, we described a nonviral cytoplasmic gene therapy vector system based on the T7 autogene concept. This system has been shown to achieve rapid and high levels of gene expression in a variety of animal cells and tissues. To test the utility of the system in vivo tumor ablation, a T7 cancer gene therapy plasmi...

journal_title：Human gene therapy

authors： Chen X,Li Y,Xiong K,Aizicovici S,Xie Y,Zhu Q,Sturtz F,Shulok J,Snodgrass R,Wagner TE,Platika D

更新日期：1998-03-20 00:00:00

abstract：:Foamy virus (FV) vectors are a promising gene delivery system for use in hematopoietic stem cell gene therapy. Previous FV vector marking studies in the NOD/SCID xenotransplantation model used umbilical cord blood (UCB)-derived SCID repopulating cells (SRCs) that were assayed 5-10 weeks posttransplantation. We now rep...

journal_title：Human gene therapy

authors： Josephson NC,Trobridge G,Russell DW

更新日期：2004-01-01 00:00:00

abstract：:Pulmonary edema is cleared via active Na(+) transport by alveolar epithelial Na(+)/K(+)-ATPases and Na(+) channels. Rats exposed to acute hyperoxia have a high mortality rate, decreased Na(+)/K(+)-ATPase function, and decreased alveolar fluid clearance (AFC). We hypothesized that Na(+)/K(+)-ATPase subunit gene overexp...

journal_title：Human gene therapy

authors： Factor P,Dumasius V,Saldias F,Brown LA,Sznajder JI

更新日期：2000-11-01 00:00:00

abstract：:Adenovirus-based vectors comprise the most frequently used vector type in clinical studies to date. Both intense lab research and insights from the clinical trials reveal the importance of a comprehensive understanding of vector-host interactions. Especially for systemic intravenous adenovirus vector delivery, it is p...

journal_title：Human gene therapy

authors： Hagedorn C,Kreppel F

更新日期：2017-10-01 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：:One of the major obstacles to pulmonary-directed gene therapy using adenoviral vectors is the induction of inflammation. We investigated whether the adenoviral particles that constitute the initial inoculum can serve as an inflammatory stimulus, independent of their ability to express genes that they contain. Viral pa...

journal_title：Human gene therapy

authors： McCoy RD,Davidson BL,Roessler BJ,Huffnagle GB,Janich SL,Laing TJ,Simon RH

更新日期：1995-12-01 00:00:00

abstract：:The expanding use of adenoviral vectors for gene therapy has brought about the need for new analytical tools. We have developed an anion-exchange high-performance liquid chromatography method to analyze recombinant adenovirus serotype 5 samples. Before this assay, available analytical methods consisted of either long-...

journal_title：Human gene therapy

authors： Shabram PW,Giroux DD,Goudreau AM,Gregory RJ,Horn MT,Huyghe BG,Liu X,Nunnally MH,Sugarman BJ,Sutjipto S

更新日期：1997-03-01 00:00:00

abstract：:Oncolytic measles virus (MV) encoding the human thyroidal sodium iodide symporter (MV-NIS) has proved to be safe after intraperitoneal or intravenous administration in patients with ovarian cancer or multiple myeloma, respectively, but it has not yet been administered through intratumoral injection in humans. Squamous...

journal_title：Human gene therapy

authors： Li H,Peng KW,Russell SJ

更新日期：2012-03-01 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

authors： Suzuki M,Singh RN,Crystal RG

更新日期：1996-10-01 00:00:00

abstract：:Gene-modified lymphocytes have a potential role in the therapy of cancer, infectious diseases, and genetic disorders of the immune system. Current gene therapy protocols involving gene transfer into lymphocytes utilize retroviruses with amphotropic envelope proteins. However, transduction efficiencies in lymphocytes u...

journal_title：Human gene therapy

authors： Lam JS,Reeves ME,Cowherd R,Rosenberg SA,Hwu P

更新日期：1996-08-01 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：: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：:To evaluate the hypothesis that innate immune mechanisms play a major role in eliminating adenovirus (Ad) vectors from the lung, the fate of adenoviral genome of an Ad vector was quantified in the first 24 h after intratracheal administration of an Ad vector coding for beta-galactosidase (beta gal) to mice. Southern a...

journal_title：Human gene therapy

authors： Worgall S,Leopold PL,Wolff G,Ferris B,Van Roijen N,Crystal RG

更新日期：1997-09-20 00:00:00

abstract：:Adeno-associated virus (AAV) vector technology is rapidly advancing and becoming not only the leading vector platform in the field of gene therapy but also a useful tool for functional genomic studies of novel proteins. As most vectors utilize constitutive promoters, this results in transgene expression during product...

journal_title：Human gene therapy

authors： Guimaro MC,Afione SA,Tanaka T,Chiorini JA

更新日期：2020-10-01 00:00:00

abstract：:Mutations in the cilia-centrosomal protein CEP290 are frequently observed in autosomal recessive childhood blindness disorder Leber congenital amaurosis (LCA). No treatment or cure currently exists for this disorder. The Cep290rd16 (retinal degeneration 16) mouse (a model of LCA) carries a mutation in the Cep290 gene....

journal_title：Human gene therapy

authors： Zhang W,Li L,Su Q,Gao G,Khanna H

更新日期：2018-01-01 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：:Aberrant JAK/STAT3 pathway has been reported to be related to hepatocellular carcinoma (HCC) in many cell lines. In this study, a double-regulated oncolytic adenovirus vector that can replicate and induce a cytopathic effect in alpha-fetoprotein (AFP)-positive HCC cell lines with p53 dysfunction was successfully const...

journal_title：Human gene therapy

authors： Wei RC,Cao X,Gui JH,Zhou XM,Zhong D,Yan QL,Huang WD,Qian QJ,Zhao FL,Liu XY

更新日期：2011-09-01 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：:We employed the hypophysectomized rats as an animal model to explore the feasibility of using genetically engineered fibroblast cells for growth hormone gene therapy. An internal ribosome entry site (IRES)-directed bicistronic retroviral vector, PSN, which contained a porcine growth hormone (pGH) cDNA at the first cis...

journal_title：Human gene therapy

authors： Chen BF,Chang WC,Chen ST,Chen DS,Hwang LH

更新日期：1995-07-01 00:00:00

abstract：:Adenovirus type 5 (Ad5) is a commonly used vector for gene therapy, but its efficacy is limited by high seroprevalence and off-target hepatic and splenic sequestration. In order to circumvent these limitations, the use of vectors derived from rare species adenoviruses is appealing. The opportunity to retarget rare spe...

journal_title：Human gene therapy

authors： Coughlan L,Uusi-Kerttula H,Ma J,Degg BP,Parker AL,Baker AH

更新日期：2014-04-01 00:00:00

abstract：:Retroviral vectors encoding glucose-responsive promoters driving furin expression may provide an amplified, glucose-regulated secretion of insulin. We constructed LhI*TFSN virus to encode a glucose-regulatable transforming growth factor alpha promoter controlling furin expression with a viral LTR promoter driving cons...

journal_title：Human gene therapy

authors： Barry SC,Ramesh N,Lejnieks D,Simonson WT,Kemper L,Lernmark A,Osborne WR

更新日期：2001-01-20 00:00:00

abstract：:Lentiviral vectors are promising tools for gene transfer into the central nervous system. We have characterized in detail transduction with human immunodeficiency virus type 1 (HIV-1)-derived vectors encoding enhanced green fluorescent protein (eGFP) in the adult mouse brain. Different brain regions such as the striat...

journal_title：Human gene therapy

authors： Baekelandt V,Claeys A,Eggermont K,Lauwers E,De Strooper B,Nuttin B,Debyser Z

更新日期：2002-05-01 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：:Replication-competent adenoviruses may provide a highly efficient means of delivering therapeutic genes to tumors. Previously, we evaluated in vitro a replication-competent adenovirus (Ad5-CD/TKrep) containing a cytosine deaminase (CD)/herpes simplex type 1 thymidine kinase (HSV-1 TK) fusion gene that allows lytic vir...

journal_title：Human gene therapy

authors： Rogulski KR,Wing MS,Paielli DL,Gilbert JD,Kim JH,Freytag SO

更新日期：2000-01-01 00:00:00

abstract：:Successful gene therapy approaches for metachromatic leukodystrophy (MLD), based either on hematopoietic stem/progenitor cells (HSPCs) or direct central nervous system (CNS) gene transfer, highlighted a requirement for high levels of arylsulfatase A (ARSA) expression to achieve correction of disease manifestations in ...

journal_title：Human gene therapy

authors： Capotondo A,Cesani M,Pepe S,Fasano S,Gregori S,Tononi L,Venneri MA,Brambilla R,Quattrini A,Ballabio A,Cosma MP,Naldini L,Biffi A

更新日期：2007-09-01 00:00:00