Successful target cell transduction of capsid-engineered rAAV vectors requires clathrin-dependent endocytosis.

abstract：:Short hairpin RNA (shRNA)-mediated gene regulation is a commonly used technique for gene manipulation. An efficient and safe delivery system is indispensable when shRNA is delivered into living organisms for gene therapy. Previous studies have proved that DNA and protein can be delivered into dendritic cells (DCs) by ...

journal_title：Gene therapy

authors： Zhang L,Zhang T,Wang L,Shao S,Chen Z,Zhang Z

更新日期：2014-07-01 00:00:00

abstract：:Cationic lipid-DNA complexes (lipoplexes) have been widely used as gene transfer vectors which avoid the adverse immunogenicity and potential for viraemia of viral vectors. With the long-term aim of gene transfer into skeletal muscle in vivo, we describe a direct in vitro comparison of two commercially available catio...

journal_title：Gene therapy

authors： Dodds E,Dunckley MG,Naujoks K,Michaelis U,Dickson G

更新日期：1998-04-01 00:00:00

abstract：:Intravenous delivery of adenoviruses is the optimal route for many gene therapy applications. Once in the blood, coagulation factor X (FX) binds to the adenovirus capsid and protects the virion from natural antibody and classical complement-mediated neutralisation in mice. However, to date, no studies have examined th...

journal_title：Gene therapy

authors： Duffy MR,Doszpoly A,Turner G,Nicklin SA,Baker AH

更新日期：2016-07-01 00:00:00

abstract：:A novel class of cationic hyperbranched polymers, containing branched oligoethylenimine (OEI 800 Da) as core, diacrylate esters as linkers and oligoamines as surface modification, was synthesized and evaluated regarding their structure-activity relationship as gene carriers. We show that pseudodendritic core character...

journal_title：Gene therapy

authors： Russ V,Elfberg H,Thoma C,Kloeckner J,Ogris M,Wagner E

更新日期：2008-01-01 00:00:00

abstract：:Gyrate atrophy (GA) of the choroid and retina is an autosomal recessive chorioretinal degeneration, caused by deficiency of the mitochondrial matrix enzyme ornithine-delta-aminotransferase (OAT). This deficiency results in the accumulation of ornithine in the body fluids and leads to hyperornithinemia. Although the cl...

journal_title：Gene therapy

authors： Lacorazza HD,Jendoubi M

更新日期：1995-01-01 00:00:00

abstract：:In 1998, two distinct and exciting scientific fields emerged which have profoundly shaped the current direction of biomedical research. The discovery of RNA interference (RNAi) and the derivation of human embryonic stem (ES) cells have yielded exciting new possibilities for researchers and clinicians alike. While fund...

journal_title：Gene therapy

authors： Heidersbach A,Gaspar-Maia A,McManus MT,Ramalho-Santos M

更新日期：2006-03-01 00:00:00

abstract：:This study was designed to determine whether Coxsackie adenovirus receptor (CAR) and alpha nu beta3/alpha nu beta5 integrin co-receptors are involved in adenovirus gene transfer in the rat cochlea. We find that CAR and integrin co-receptors are expressed in every cell subtype transduced by the adenoviral vector Ad5 De...

journal_title：Gene therapy

authors： Venail F,Wang J,Ruel J,Ballana E,Rebillard G,Eybalin M,Arbones M,Bosch A,Puel JL

更新日期：2007-01-01 00:00:00

abstract：:Integration site analysis was performed on six dogs with canine leukocyte adhesion deficiency (CLAD) that survived greater than 1 year after infusion of autologous CD34+ bone marrow cells transduced with a gammaretroviral vector expressing canine CD18. A total of 387 retroviral insertion sites (RIS) were identified in...

journal_title：Gene therapy

authors： Hai M,Adler RL,Bauer TR Jr,Tuschong LM,Gu YC,Wu X,Hickstein DD

更新日期：2008-07-01 00:00:00

abstract：:Transplantation of allogeneic pancreatic islets is an effective approach to treat type 1 diabetes. To bypass the need for systemic administration of immunosuppression drugs following transplantation, approaches to genetically modify allogeneic islets to express anti-inflammatory, immunosuppressive, or antiapoptotic pr...

journal_title：Gene therapy

authors： Rehman KK,Wang Z,Bottino R,Balamurugan AN,Trucco M,Li J,Xiao X,Robbins PD

更新日期：2005-09-01 00:00:00

abstract：:To induce RNA interference (RNAi), either small interfering RNAs (siRNAs) are directly introduced into the cell or short hairpin RNAs (shRNAs) are expressed from a DNA vector. At present, shRNAs are commonly synthesized by RNA polymerase III (Pol III) promoters of the H1 and U6 RNAs. In this study, we designed and cha...

journal_title：Gene therapy

authors： Choy EY,Kok KH,Tsao SW,Jin DY

更新日期：2008-02-01 00:00:00

abstract：:We previously demonstrated that recombinant adeno-associated virus vectors based on serotype 2 (rAAV2) can direct transgene expression in salivary gland cells in vitro and in vivo. However, it is not known how other rAAV serotypes perform when infused into salivary glands. The capsids of serotypes 4 and 5 are distinct...

journal_title：Gene therapy

authors： Katano H,Kok MR,Cotrim AP,Yamano S,Schmidt M,Afione S,Baum BJ,Chiorini JA

更新日期：2006-04-01 00:00:00

abstract：:Inhibition of virus replication by means of RNA interference has been reported for several important human pathogens, including human immunodeficiency virus type 1 (HIV-1). RNA interference against these pathogens has been accomplished by introduction of virus-specific synthetic small interfering RNAs (siRNAs) or DNA ...

journal_title：Gene therapy

authors： Konstantinova P,de Vries W,Haasnoot J,ter Brake O,de Haan P,Berkhout B

更新日期：2006-10-01 00:00:00

abstract：:Numerous approaches in gene therapy of human cancers are focused on the establishment of cell type specific or inducible expression vectors allowing the targeted and regulated expression of therapeutic genes. Various conditionally active vectors have been created carrying promoters responding to certain factors or the...

journal_title：Gene therapy

authors： Walther W,Wendt J,Stein U

更新日期：1997-06-01 00:00:00

abstract：:The ability to express recombinant genes in the coronary vasculature and the myocardium holds promise for the treatment of a number of acquired and inherited cardiovascular diseases. Previous in vivo gene transfer approaches in the heart have been limited by relatively low efficiencies of gene transduction. In this re...

journal_title：Gene therapy

authors： Barr E,Carroll J,Kalynych AM,Tripathy SK,Kozarsky K,Wilson JM,Leiden JM

更新日期：1994-01-01 00:00:00

abstract：:Breast cancer is the leading cancer diagnosed in women and the second leading cause of cancer-related deaths in women. Current limitations to standard chemotherapy in the clinic are extensively researched, including problems arising from repeated treatments with the same drugs. The phenomenon that cancer cells become ...

journal_title：Gene therapy

authors： Jones SK,Merkel OM

更新日期：2016-12-01 00:00:00

abstract：:Signal transducer and activator of transcription 3 (STAT3) is constitutively activated in diverse cancers, which contributes to the proliferation and survival of cancer cells by upregulating apoptosis inhibitors and cell cycle regulators. Suppressor of cytokine signaling 1 (SOCS1) is an important negative regulator of...

journal_title：Gene therapy

authors： Liu L,Li W,Wei X,Cui Q,Lou W,Wang G,Hu X,Qian C

更新日期：2013-01-01 00:00:00

abstract：:Fibrodysplasia ossificans progressiva (FOP) is an autosomal dominant congenital disorder characterized by progressive heterotopic bone formation. Currently, no definitive treatment exists for FOP. The activin receptor type IA / activin-like kinase 2 (ACVR1/ALK2) gene has been identified as the responsible gene for FOP...

journal_title：Gene therapy

authors： Takahashi M,Katagiri T,Furuya H,Hohjoh H

更新日期：2012-07-01 00:00:00

abstract：:Gene therapy is thought to be a promising method for the treatment of various diseases. One gene therapy strategy involves the manipulations on a process of formation of new vessels, commonly defined as angiogenesis. Angiogenic and antiangiogenic gene therapy is a new therapeutic approach to the treatment of cardiovas...

journal_title：Gene therapy

authors： Malecki M,Kolsut P,Proczka R

更新日期：2005-10-01 00:00:00

abstract：:We introduce an injectable system for the formation of a biodegradable DNA-containing implant that releases DNA over a 2-month period to provide a robust and prolonged gene expression at the site. Sustained delivery of the appropriate plasmid DNA resulted in sustained expression of luciferase, the persistent appearanc...

journal_title：Gene therapy

authors： Eliaz RE,Szoka FC Jr

更新日期：2002-09-01 00:00:00

abstract：:Silencing of gene expression by small interfering RNAs (siRNAs) is rapidly becoming a powerful tool for genetic analysis of mammalian cells. The use of DNA-based plasmid vectors to achieve transient and stable expression of siRNA has been developed to avoid the problems of double-stranded oligonucleotides transfection...

journal_title：Gene therapy

authors： Sabbioni S,Callegari E,Manservigi M,Argnani R,Corallini A,Negrini M,Manservigi R

更新日期：2007-03-01 00:00:00

abstract：:Insulin-like growth factor-I (IGF-I) and its receptor (IGF-IR) are essential for normal ocular development and are expressed in numerous ocular cell types including lens epithelial cells, retinal pigment epithelial cells, Müller cells and endothelial cells. Endothelial cell proliferation is a common feature of prolife...

journal_title：Gene therapy

authors： Shaw LC,Pan H,Afzal A,Calzi SL,Spoerri PE,Sullivan SM,Grant MB

更新日期：2006-05-01 00:00:00

abstract：:The mechanism whereby cationic lipids destabilize cell membranes to facilitate the intracellular delivery of macromolecules such as plasmid DNA or antisense oligonucleotides is not well understood. Here, we show that cationic lipids can destabilize lipid bilayers by promoting the formation of nonbilayer lipid structur...

journal_title：Gene therapy

authors： Hafez IM,Maurer N,Cullis PR

更新日期：2001-08-01 00:00:00

abstract：:For patients with metastatic prostate cancer, the 5-year survival rate of 31% points to a need for novel therapies and improvement of existing modalities. We propose that p53 gene therapy and chemotherapy, when combined, will provide superior tumor cell killing for the treatment of prostate carcinoma. To this end, we ...

journal_title：Gene therapy

authors： Tamura RE,Lana MG,Costanzi-Strauss E,Strauss BE

更新日期：2020-02-01 00:00:00

abstract：:For many gene therapy applications the effective titre of retroviral vectors is a limiting factor both in vitro and in vivo. Purification and concentration of retrovirus from packaging cell supernatant can overcome this problem. To this end we have investigated a novel procedure which involves complexing retrovirus to...

journal_title：Gene therapy

authors： Darling D,Hughes C,Galea-Lauri J,Gäken J,Trayner ID,Kuiper M,Farzaneh F

更新日期：2000-06-01 00:00:00

abstract：:Mesenchymal stem cells (MSC) are a group of clonogenic cells present among the bone marrow stroma and capable of multilineage differentiation into mesoderm-type cells such as osteoblasts, adipocytes and chondrocytes. Due to their ease of isolation and their differentiation potential, MSC are being introduced into clin...

journal_title：Gene therapy

authors： Abdallah BM,Kassem M

更新日期：2008-01-01 00:00:00

abstract：:Newcastle disease virus (NDV) is an oncolytic paramyxovirus with a nonsegmented single-stranded RNA genome. In this report, a recombinant oncolytic NDV was passaged in human tumor xenografts and reisolated and characterized after two rounds of bioselection. Several isolates could be recovered that differed from the pa...

journal_title：Gene therapy

authors： Beier R,Hermiston T,Mumberg D

更新日期：2013-01-01 00:00:00

abstract：:Exogenous osteoprotegerin (OPG) gene modification appears a therapeutic strategy for osteolytic aseptic loosening. The feasibility and efficacy of a cell-based OPG gene delivery approach were investigated using a murine model of knee prosthesis failure. A titanium pin was implanted into mouse proximal tibia to mimic a...

journal_title：Gene therapy

authors： Zhang L,Jia TH,Chong AC,Bai L,Yu H,Gong W,Wooley PH,Yang SY

更新日期：2010-10-01 00:00:00

abstract：:In this study, self-inactivating (SIN) retroviral vectors based on feline foamy virus (FFV) were constructed and analysed. The FFV SIN vectors were devoid of the core FFV long terminal repeat promoter plus upstream sequences but contained all structural and regulatory genes. This design allowed sensitive detection of ...

journal_title：Gene therapy

authors： Bastone P,Löchelt M

更新日期：2004-03-01 00:00:00

abstract：:Plasmids carrying the Epstein-Barr virus (EBV) latent gene EBNA1 and the EBV latent origin of replication (oriP) stay in transfected human cells as autonomously replicating extrachromosomal genetic units. They thus might represent a suitable tool for cytokine gene introduction into human tumor cells with the prospect ...

journal_title：Gene therapy

authors： Mũcke S,Polack A,Pawlita M,Zehnpfennig D,Massoudi N,Bohlen H,Doerfler W,Bornkamm G,Diehl V,Wolf J

更新日期：1997-02-01 00:00:00

abstract：:A replication-incompetent adenoviral vector encoding the heavy chain C-fragment (H(C)50) of botulinum neurotoxin type C (BoNT/C) was evaluated as a mucosal vaccine against botulism in a mouse model. Single intranasal inoculation of the adenoviral vector elicited a high level of H(C)50-specific IgG, IgG1 and IgG2a in s...

journal_title：Gene therapy

authors： Xu Q,Pichichero ME,Simpson LL,Elias M,Smith LA,Zeng M

更新日期：2009-03-01 00:00:00