Efficient bone marrow transduction by gene transfer with allogeneic umbilical cord blood serum and plasma: an implication for clinical trials.

abstract：:The liver is an attractive target tissue for gene therapy. Current approaches for hepatic gene delivery include retroviral and adenoviral vectors, liposome/DNA, and peptide/DNA complexes. This study describes a technique for direct injection of DNA into liver that led to significant gene expression. Gene expression wa...

journal_title：Human gene therapy

authors： Hickman MA,Malone RW,Lehmann-Bruinsma K,Sih TR,Knoell D,Szoka FC,Walzem R,Carlson DM,Powell JS

更新日期：1994-12-01 00:00:00

abstract：:Leukocyte adhesion deficiency type I (LAD-I) is a primary immunodeficiency caused by mutations in the ITGB2 gene and is characterized by recurrent and life-threatening bacterial infections. These mutations lead to defective or absent expression of β2 integrins on the leukocyte surface, compromising adhesion and extrav...

journal_title：Human gene therapy

authors： Leon-Rico D,Aldea M,Sanchez-Baltasar R,Mesa-Nuñez C,Record J,Burns SO,Santilli G,Thrasher AJ,Bueren JA,Almarza E

更新日期：2016-09-01 00:00:00

abstract：:Recent advances in genome sequencing have greatly improved our ability to understand and identify the causes of genetic diseases. However, there remains an urgent need for innovative, safe, and effective treatments for these diseases. CRISPR-based genome editing systems have become important and powerful tools in the ...

journal_title：Human gene therapy

authors： Carlaw TM,Zhang LH,Ross CJD

更新日期：2020-08-01 00:00:00

abstract：:Human adenoviruses are the most widely used vectors in gene medicine, with applications ranging from oncolytic therapies to vaccinations, but adenovirus vectors are not without side effects. In addition, natural adenoviruses pose severe risks for immunocompromised people, yet infections are usually mild and self-limit...

journal_title：Human gene therapy

authors： Hendrickx R,Stichling N,Koelen J,Kuryk L,Lipiec A,Greber UF

更新日期：2014-04-01 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：: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：: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：:Direct arterial gene transfer has been previously achieved using double-balloon catheters and perforated balloons, in most cases facilitated by the use of cationic liposomes or viral vectors. These gene delivery systems, however, have been compromised by issues relating to efficacy and/or safety, and furthermore requi...

journal_title：Human gene therapy

authors： Riessen R,Rahimizadeh H,Blessing E,Takeshita S,Barry JJ,Isner JM

更新日期：1993-12-01 00:00:00

abstract：:Hematopoietic stem cells (HSCs) are a potential target for the retrovirus-mediated transfer of chemotherapeutic drug resistance genes. For integration of the proviral DNA in the HSC genome cell division is required. In the bone marrow (BM) hematopoiesis occurs in the vicinity of stroma cells. Soluble stroma components...

journal_title：Human gene therapy

authors： Schiedlmeier B,Buss EC,Veldwijk MR,Zeller WJ,Fruehauf S

更新日期：1999-06-10 00:00:00

abstract：:Adoptive cellular therapy provides the promise of a potentially powerful general treatment for cancer. Although this is a complex and challenging field, there have been major advances in basic and translational research resulting in clinical trial activity that is now beginning to confirm this promise. However, these ...

journal_title：Human gene therapy

authors： Hawkins RE,Gilham DE,Debets R,Eshhar Z,Taylor N,Abken H,Schumacher TN,ATTACK Consortium

更新日期：2010-06-01 00:00:00

abstract：:Engineering gene therapy vectors to modulate the immune response is an important goal. In this regard, costimulation of T cells is a critical determinant in immune activation. The costimulatory molecule CD40, expressed on antigen-presenting cells, is thought to interact with CD40 ligand (CD40L) expressed on activated ...

journal_title：Human gene therapy

authors： Sin JI,Kim JJ,Zhang D,Weiner DB

更新日期：2001-06-10 00:00:00

abstract：:Skeletal muscle represents an attractive target tissue for adenoviral gene therapy to treat muscle disorders and as a production platform for systemic expression of therapeutic proteins. However, adenovirus serotype 5 vectors do not efficiently transduce adult muscle tissue. Here we evaluated whether capsid modificati...

journal_title：Human gene therapy

authors： Guse K,Suzuki M,Sule G,Bertin TK,Tyynismaa H,Ahola-Erkkilä S,Palmer D,Suomalainen A,Ng P,Cerullo V,Hemminki A,Lee B

更新日期：2012-10-01 00:00:00

abstract：:Gene electrotransfer is gaining momentum as an efficient methodology for nonviral gene transfer. In skeletal muscle, data suggest that electric pulses play two roles: structurally permeabilizing the muscle fibers and electrophoretically supporting the migration of DNA toward or across the permeabilized membrane. To in...

journal_title：Human gene therapy

authors： André FM,Gehl J,Sersa G,Préat V,Hojman P,Eriksen J,Golzio M,Cemazar M,Pavselj N,Rols MP,Miklavcic D,Neumann E,Teissié J,Mir LM

更新日期：2008-11-01 00:00:00

abstract：:The practical application of gene transfer as a treatment for genetic diseases such as cystic fibrosis or hemophilia has been hindered, in part, by low efficiencies of vector delivery and transgene expression. We demonstrated that a feline immunodeficiency virus (FIV)-based lentiviral vector pseudotyped with the envel...

journal_title：Human gene therapy

authors： Sinn PL,Goreham-Voss JD,Arias AC,Hickey MA,Maury W,Chikkanna-Gowda CP,McCray PB Jr

更新日期：2007-12-01 00:00:00

abstract：:Stereotactic inoculation of a herpes simplex virus (HSV) gene transfer vector into the hippocampus and caudate of rat brain resulted in limited and transient viral replication and the establishment of latency. Virus attenuation was achieved by insertional inactivation of a viral gene, Us3. Insertion of a lacZ reporter...

journal_title：Human gene therapy

authors： Fink DJ,Sternberg LR,Weber PC,Mata M,Goins WF,Glorioso JC

更新日期：1992-02-01 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：:In vivo electroporation of plasmid DNA (DNA-EP) is an efficient and safe method for vaccines. It results in increased DNA uptake, enhances protein expression, and augments immune responses to the target antigen in a variety of species. To further improve the efficacy of DNA-EP, we evaluated small interfering RNA (siRN...

journal_title：Human gene therapy

authors： Dharmapuri S,Aurisicchio L,Biondo A,Welsh N,Ciliberto G,La Monica N

更新日期：2009-06-01 00:00:00

abstract：:Vectors derived from the human parvovirus AAV-2 (adeno-associated virus type 2) are among the most promising gene delivery vehicles currently being developed. These vectors are not only capable of transducing a large variety of human cell types in vitro and in vivo, but in immunocompetent animal models can establish l...

journal_title：Human gene therapy

authors： Grimm D,Kleinschmidt JA

更新日期：1999-10-10 00:00:00

abstract：:Mutations in the gene encoding the peroxisomal ATP-binding cassette transporter (ABCD1) cause elevations in very long-chain fatty acids (VLCFAs) and the neurodegenerative disease adrenoleukodystrophy (ALD). In most adults, this manifests as the spinal cord axonopathy adrenomyeloneuropathy (AMN). A challenge in virus-b...

journal_title：Human gene therapy

authors： Gong Y,Berenson A,Laheji F,Gao G,Wang D,Ng C,Volak A,Kok R,Kreouzis V,Dijkstra IM,Kemp S,Maguire CA,Eichler F

更新日期：2019-05-01 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：: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 develop a potential gene therapy strategy for the treatment of hemophilia A, we constructed several retroviral vectors expressing a B-domain-deleted factor VIII (FVIII) cDNA. We confirmed previous reports that when the FVIII cDNA is inserted into a retroviral vector, the vector mRNA is decreased resulting in signif...

journal_title：Human gene therapy

authors： Chuah MK,VandenDriessche T,Morgan RA

更新日期：1995-11-01 00:00:00

abstract：:Advanced prostate cancer is invariably lethal once it becomes androgen independent (AI). With the aim of developing a new treatment we have used the human androgen-independent prostate cancer cell line, PC-3, to evaluate the effectiveness of two enzyme-directed prodrug therapy (EPT) systems as a novel means for promot...

journal_title：Human gene therapy

authors： Martiniello-Wilks R,Garcia-Aragon J,Daja MM,Russell P,Both GW,Molloy PL,Lockett LJ,Russell PJ

更新日期：1998-07-20 00:00:00

abstract：:Lentiviral vectors are efficiently pseudotyped with RD114-TR, a chimeric envelope glycoprotein made of the extracellular and transmembrane domains of the feline leukemia virus RD114 and the cytoplasmic tail of the murine leukemia virus amphotropic envelope. RD114-TR-pseudotyped vectors may be concentrated by centrifug...

journal_title：Human gene therapy

authors： Di Nunzio F,Piovani B,Cosset FL,Mavilio F,Stornaiuolo A

更新日期：2007-09-01 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：: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：: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：:The recombinant adenoviral (Ad) vector is being considered as a cancer vaccine platform because it efficiently induces immune responses to tumor antigens by intradermal immunization. The aims of this study were to evaluate the potential toxicities and biodistribution after a single dose or six weekly intradermal doses...

journal_title：Human gene therapy

authors： Plog MS,Guyre CA,Roberts BL,Goldberg M,St George JA,Perricone MA

更新日期：2006-07-01 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：:SV40 is an attractive potential vector with high-efficiency gene transfer into a wide variety of human tissues, including the bone marrow, a critical target organ for the cure of many diseases. In the present study, the three SV40 capsid proteins, VP1, VP2, and VP3, were produced in Spodoptera frugiperda (Sf9) insect ...

journal_title：Human gene therapy

authors： Sandalon Z,Dalyot-Herman N,Oppenheim AB,Oppenheim A

更新日期：1997-05-01 00:00:00