A controlled, Phase 1 clinical trial to evaluate the safety and effects in HIV-1 infected humans of autologous lymphocytes transduced with a ribozyme that cleaves HIV-1 RNA.

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：:Production of clinical-grade gammaretroviral vectors for ex vivo gene delivery requires a scalable process that can rapidly generate large amounts of vector supernatant, clear large numbers of residual packaging cells with minimal decreases in vector titer, and satisfy all current regulatory guidelines regarding produ...

journal_title：Human gene therapy

authors： Feldman SA,Goff SL,Xu H,Black MA,Kochenderfer JN,Johnson LA,Yang JC,Wang Q,Parkhurst MR,Cross S,Morgan RA,Cornetta K,Rosenberg SA

更新日期：2011-01-01 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：: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：: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：:Adenovirus vectors transduce liver hepatocytes with extreme efficiency; however, transgene expression is eliminated within 2 weeks. Extinction of transgene expression has been attributed to infiltrating cytotoxic T lymphocytes (CTLs) in the liver in a process that resembles a number of human diseases, including viral ...

journal_title：Human gene therapy

authors： Chirmule N,Moscioni AD,Qian Y,Qian R,Chen Y,Wilson JM

更新日期：1999-01-20 00:00:00

abstract：:As efficient and less toxic virus-derived gene therapy vectors are developed, a pressing problem is to avoid immune response to the therapeutic gene product. Secreted therapeutic proteins potentially represent a special problem, as they are readily available to professional antigen-presenting cells throughout the body...

journal_title：Human gene therapy

authors： Cerullo V,McCormack W,Seiler M,Mane V,Cela R,Clarke C,Rodgers JR,Lee B

更新日期：2007-12-01 00:00:00

abstract：:This is an erratum of the published paper "Preclinical Evaluation of Chimeric Antigen Receptor-Modified T Cells Specific to Epithelial Cell Adhesion Molecule for Treating Colorectal Cancer". There are some errors in figure 6C and 7C in the article due to authors' mistakes when preparing the figures. Specifically, repr...

journal_title：Human gene therapy

authors： Wang W

更新日期：2019-08-01 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：:Human embryonic stem cells (hESC) and induced pluripotent stem cells (iPSC) offer great hope for in vitro modeling of Parkinson's disease (PD), as well as for designing cell-replacement therapies. To realize these opportunities, there is an urgent need to develop efficient protocols for the directed differentiation of...

journal_title：Human gene therapy

authors： Sánchez-Danés A,Consiglio A,Richaud Y,Rodríguez-Pizà I,Dehay B,Edel M,Bové J,Memo M,Vila M,Raya A,Izpisua Belmonte JC

更新日期：2012-01-01 00:00:00

abstract：:Gutted adenoviral (Ad) vectors have a greater cloning capacity and elicit less immune response than conventional Ad vectors. Unfortunately, clinical use of gutted vectors has been slowed by production difficulties, including low yield and a tendency for recombinant virus to emerge. These two problems are related, beca...

journal_title：Human gene therapy

authors： Hartigan-O'Connor D,Barjot C,Crawford R,Chamberlain JS

更新日期：2002-03-01 00:00:00

abstract：:In summary, I will reiterate the five points I would like to leave with you today: First, the biological revolution has extraordinary power to do good. As long as the use of our new genetic knowledge is guided by the traditional ideals of the healing professions--to help improve the human condition without doing harm-...

journal_title：Human gene therapy

authors： Healy B

更新日期：1992-02-01 00:00:00

abstract：:Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene that result in the absence of functional protein. In the majority of cases these are out-of-frame deletions that disrupt the reading frame. Several attempts have been made to restore the dystrophin mRNA reading frame by modulation of pre-m...

journal_title：Human gene therapy

authors： Arechavala-Gomeza V,Graham IR,Popplewell LJ,Adams AM,Aartsma-Rus A,Kinali M,Morgan JE,van Deutekom JC,Wilton SD,Dickson G,Muntoni F

更新日期：2007-09-01 00:00:00

abstract：:Isolated methylmalonic acidemia (MMA), a group of autosomal recessive inborn errors of metabolism, is most commonly caused by complete (mut(0)) or partial (mut(-)) deficiency of the enzyme methylmalonyl-CoA mutase (MUT). The severe metabolic instability and increased mortality experienced by many affected individuals,...

journal_title：Human gene therapy

authors： Harrington EA,Sloan JL,Manoli I,Chandler RJ,Schneider M,McGuire PJ,Calcedo R,Wilson JM,Venditti CP

更新日期：2016-05-01 00:00:00

abstract：:Genetic immunization has been widely applied in efforts to find novel and efficient mechanisms of stimulating the immune response. An effective attack against viral pathogens or tumors often requires activation of T cell-mediated immunity and the generation of cytotoxic T cells. Intramuscular immunization with plasmid...

journal_title：Human gene therapy

authors： Hedley ML,Strominger JL,Urban RG

更新日期：1998-02-10 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：:Retinal ganglion cells (RGCs) play a key role in the pathogenesis and development of glaucoma. The present study aims to investigate the underlying mechanism of long noncoding RNA growth arrest-specific transcript 5 (GAS5) in glaucoma development through regulating the apoptosis of RGCs. Rat models of chronic glaucoma...

journal_title：Human gene therapy

authors： Zhou RR,Li HB,You QS,Rong R,You ML,Xiong K,Huang JF,Xia XB,Ji D

更新日期：2019-12-01 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：:Despite improvements in drug and device therapy for heart failure, hospitalization rates and mortality have changed little in the past decade. Randomized clinical trials using gene transfer to improve function of the failing heart are the focus of this review. Four randomized clinical trials of gene transfer in heart ...

journal_title：Human gene therapy

authors： Penny WF,Hammond HK

更新日期：2017-05-01 00:00:00

abstract：:The influence of serum on the production of retroviral vectors by the HT1080 human fibrosarcoma-derived packaging cell line FLYRD18 was investigated. A fourfold increase in virus titer was observed under serum-free conditions, as compared with medium supplemented with 10% fetal calf serum. A similar improvement was al...

journal_title：Human gene therapy

authors： Gerin PA,Gilligan MG,Searle PF,Al-Rubeai M

更新日期：1999-08-10 00:00:00

abstract：:A human immunodeficiency virus type 1 (HIV-1)-based retroviral vector pseudotyped with HIV envelope containing the herpes simplex virus-thymidine kinase (HSV-TK) gene under the control of the HIV LTR promoter (pHXTKN) was constructed and stably transferred into human CD4(+) H9, CEM, and U937 cells. RNase protection as...

journal_title：Human gene therapy

authors： Miyake K,Iijima O,Suzuki N,Matsukura M,Shimada T

更新日期：2001-02-10 00:00:00

abstract：:Duchenne muscular dystrophy (DMD) is a lethal genetic disorder for which there is currently no effective treatment. Although clinical application of adenoviral vector-mediated gene transfer has not been fully developed, it shows promise for the treatment of DMD. One significant problem posed by adenoviral vector-media...

journal_title：Human gene therapy

authors： Clemens PR,Krause TL,Chan S,Korb KE,Graham FL,Caskey CT

更新日期：1995-11-01 00:00:00

abstract：:Lentiviral vectors are efficient gene delivery vehicles for therapeutic and research applications. In contrast to oncoretroviral vectors, they are able to infect most nonproliferating cells. In the liver, induction of cell proliferation dramatically improved hepatocyte transduction using all types of retroviral vector...

journal_title：Human gene therapy

authors： Pichard V,Couton D,Desdouets C,Ferry N

更新日期：2013-02-01 00:00:00

abstract：:Human immunodeficiency virus (HIV) infection represents one of the most challenging systems for gene therapy. Thanks to the extended knowledge of the molecular biology of the HIV life cycle, many different strategies have been developed including transdominant modifications of HIV proteins, RNA decoys, antisense RNA, ...

journal_title：Human gene therapy

authors： Michienzi A,Conti L,Varano B,Prislei S,Gessani S,Bozzoni I

更新日期：1998-03-20 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：: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：:We evaluated the ability of a replication-deficient, recombinant adenoviral vector to transfer the bifunctional gene GAL-TEK, which expresses a marking/therapeutic gene product, to naturally occurring cat fibrosarcomas in situ. GAL-TEK contains an in-frame fusion of the bacterial LacZ gene for histochemical marking of...

journal_title：Human gene therapy

authors： Marini FC 3rd,Cannon JP,Belmont JW,Shillitoe EJ,Lapeyre JN

更新日期：1995-09-01 00:00:00

abstract：:Recombinant adeno-associated virus (rAAV) is a prototypical gene therapy vector characterized by excellent safety profiles, wide host range, and the ability to transduce differentiated cells. Numerous rAAV-based vectors providing efficient and sustained expression of transgenes in target tissues have been developed fo...

journal_title：Human gene therapy

authors： Zolotukhin S

更新日期：2005-05-01 00:00:00

abstract：:Genetically modified lymphoblastoid cell lines (LCL) have been shown to be an attractive alternative source of antigen-presenting cells for cancer vaccination in vitro. We tested their application in patients with pancreatic cancer in a phase I clinical trial. As a model tumor antigen, we selected the point-mutated (c...

journal_title：Human gene therapy

authors： Kubuschok B,Pfreundschuh M,Breit R,Hartmann F,Sester M,Gärtner B,König J,Murawski N,Held G,Zwick C,Neumann F

更新日期：2012-12-01 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