New frontier in regenerative medicine: site-specific gene correction in patient-specific induced pluripotent stem cells.

abstract：:RNA interference (RNAi) is a form of posttranscriptional gene silencing mediated by short double-stranded RNA, known as small interfering RNA (siRNA). These siRNAs are capable of binding to a specific mRNA sequence and causing its degradation. The recent demonstration of a plasmid vector that directs siRNA synthesis i...

journal_title：Human gene therapy

authors： Abbas-Terki T,Blanco-Bose W,Déglon N,Pralong W,Aebischer P

更新日期：2002-12-10 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：:Autoimmune destruction of islets in the pancreas leads to the development of insulin-dependent diabetes mellitus (IDDM). Replacement of insulin-producing tissue by transplantation of islets provides a cure to disease but requires immunosuppression or a means of controlling anti-graft immune responses. To promote islet...

journal_title：Human gene therapy

authors： Gallichan WS,Kafri T,Krahl T,Verma IM,Sarvetnick N

更新日期：1998-12-10 00:00:00

abstract：:Classical gene therapy for cystic fibrosis has had limited success because of immune response against viral vectors and short-term expression of cDNA-based transgenes. These limitations could be overcome by delivering the complete genomic CFTR gene on nonintegrating human artificial chromosomes (HACs). Here, we report...

journal_title：Human gene therapy

authors： Rocchi L,Braz C,Cattani S,Ramalho A,Christan S,Edlinger M,Ascenzioni F,Laner A,Kraner S,Amaral M,Schindelhauer D

更新日期：2010-09-01 00:00:00

abstract：:Gyrate atrophy is a progressive blindness associated with deficiency of ornithine aminotransferase (OAT). The strategy of using an autologous keratinocyte graft, modified to express high levels of OAT as an ornithine-catabolizing skin-based enzyme sink, is investigated. Two OAT-containing retroviral vectors were const...

journal_title：Human gene therapy

authors： Jensen TG,Sullivan DM,Morgan RA,Taichman LB,Nussenblatt RB,Blaese RM,Csaky KG

更新日期：1997-11-20 00:00:00

abstract：:Gene transfer for therapeutic angiogenesis represents a novel treatment for patients with chronic angina refractory to standard medical therapy and not amenable to conventional revascularization. We sought to assess the role of intraoperative multiplane transesophageal echocardiography (MPTEE) in guiding injection of ...

journal_title：Human gene therapy

authors： Esakof DD,Maysky M,Losordo DW,Vale PR,Lathi K,Pastore JO,Symes JF,Isner JM

更新日期：1999-09-20 00:00:00

abstract：:DNA vaccination is an attractive approach for tumor immunotherapy because of its stability and simplicity of delivery. Advances demonstrate that helper T cell responses play a critical role in initiating immune responses. The aim of the current study is to test whether targeting HPV-16 E7 to the endosomal/lysosomal co...

journal_title：Human gene therapy

authors： Ji H,Wang TL,Chen CH,Pai SI,Hung CF,Lin KY,Kurman RJ,Pardoll DM,Wu TC

更新日期：1999-11-20 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

abstract：:Abstract Malignant gliomas (MGs) are highly vascularized, aggressive brain cancers carrying a dismal prognosis. Because of their high vascularity, anti-angiogenic therapy is a potential treatment option. Indeed, the anti-vascular endothelial growth factor (VEGF) antibody bevacizumab has demonstrated promising results ...

journal_title：Human gene therapy

authors： Samaranayake HD,Pikkarainen JT,Wirth T,Stedt H,Lesch HP,Dragneva G,Vuorio T,Määttä AM,Airenne K,Ylä-Herttuala S

更新日期：2014-11-01 00:00:00

abstract：:Kallistatin is a serine proteinase inhibitor that has been shown to reduce joint swelling and to inhibit inflammation in a rat model of arthritis. In this study, we investigated the effect and mechanisms of kallistatin on cardiac function after myocardial ischemia-reperfusion (I/R) injury. The human kallistatin gene i...

journal_title：Human gene therapy

authors： Chao J,Yin H,Yao YY,Shen B,Smith RS Jr,Chao L

更新日期：2006-12-01 00:00:00

abstract：:Low in vivo transduction efficiency and safety concerns have been hurdles for effective hematopoietic stem cell (HSC) gene therapy. Here, we investigate whether the safety and efficiency of retroviral gene transfer into HSCs can be improved by using human allogeneic umbilical cord blood (UCB)-derived supplements inste...

journal_title：Human gene therapy

authors： Moon N,Yang SJ,Park BB,Chung YS,Lee JW,Oh IH

更新日期：2008-07-01 00:00:00

abstract：:Oncolytic viruses (OV) are novel cancer gene therapies that are moving toward the forefront of modern medicines. However, their full therapeutic potential is hindered by the lack of convenient and reliable strategies to visualize and quantify OV growth kinetics and therapeutic efficacy in live cells. Here, we present ...

journal_title：Human gene therapy

authors： Quillien L,Top S,Kappler S,Redouté A,Dusetti N,Quentin-Froignant C,Lulka H,Camus C,Buscail L,Gallardo F,Bertagnoli S,Cordelier P

更新日期：2021-01-27 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：:Sphingosine kinase 1 (SPK1) has been identified as a central mediator of ischemia preconditioning and plays a protective role in ischemia/reperfusion (I/R)-induced cardiomyocyte death. In the present study, we investigated the protective effect of adenovirus-mediated SPK1 gene (Ad-SPK1) transfer on I/R-induced cardiac...

journal_title：Human gene therapy

authors： Duan HF,Wang H,Yi J,Liu HJ,Zhang QW,Li LB,Zhang T,Lu Y,Wu CT,Wang LS

更新日期：2007-11-01 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：:Blood vessels are among the easiest targets for gene therapy. However, no data are available about the safety and feasibility of intracoronary gene transfer in humans. We studied the safety and efficacy of catheter-mediated vascular endothelial growth factor (VEGF) plasmid/liposome (P/L) gene transfer in human coronar...

journal_title：Human gene therapy

authors： Laitinen M,Hartikainen J,Hiltunen MO,Eränen J,Kiviniemi M,Närvänen O,Mäkinen K,Manninen H,Syvänne M,Martin JF,Laakso M,Ylä-Herttuala S

更新日期：2000-01-20 00:00:00

abstract：:Based on the K8/JTS-1-mediated transfection technique, we developed an in vivo protocol for an efficient transfer of plasmid DNA to ocular cells. As determined with condensed plasmids containing reporter genes for either beta-galactosidase (pcDNA-lacZ) or enhanced green fluorescent protein (pREP-EGFP), the immortalize...

journal_title：Human gene therapy

authors： Neuner-Jehle M,Berghe LV,Bonnel S,Uteza Y,Benmeziane F,Rouillot JS,Marchant D,Kobetz A,Dufier JL,Menasche M,Abitbol M

更新日期：2000-09-01 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：: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：:NKG2D ligands (NKG2DLs) are widely expressed on ovarian cancers to various degrees, making them attractive targets for immunotherapy. Here, we applied a chimeric antigen receptor (CAR) approach for the targeting of NKG2DLs expressed on human ovarian cancer cells and evaluated the impact of pharmacological upregulation...

journal_title：Human gene therapy

authors： Song DG,Ye Q,Santoro S,Fang C,Best A,Powell DJ Jr

更新日期：2013-03-01 00:00:00

abstract：:An E1-, E2a-, E3-deleted adenoviral vector (Av3H82) encoding an epitope-tagged B domain-deleted human factor VIII cDNA (flagged FVIII) was evaluated in nonhuman primates. Twelve cynomolgus monkeys received intravenous administration of Av3H82; 6 monkeys received 6 x 10(11) particles/kg and another 6 received 3 x 10(12...

journal_title：Human gene therapy

authors： Brann T,Kayda D,Lyons RM,Shirley P,Roy S,Kaleko M,Smith T

更新日期：1999-12-10 00:00:00

abstract：:Targeted therapy produces objective responses in bladder cancer patients, although the responses can be short. Meanwhile, response rates to immune therapy are lower, but the effects are more durable. Based on these findings, it was hypothesized that urothelial carcinoma associated 1 (UCA1)-targeted therapy could syner...

journal_title：Human gene therapy

authors： Zhen S,Lu J,Chen W,Zhao L,Li X

更新日期：2018-12-01 00:00:00

abstract：:Myelosuppression is the main side effect of cancer chemotherapy. An improved rate of retroviral vector-mediated gene transfer to hematopoietic stem cells, shown in more recent clinical trials, has created the basis to test the concept of myeloprotective gene therapy. We transplanted clinical-scale human peripheral blo...

journal_title：Human gene therapy

authors： Schiedlmeier B,Schilz AJ,Kühlcke K,Laufs S,Baum C,Zeller WJ,Eckert HG,Fruehauf S

更新日期：2002-01-20 00:00:00

abstract：:Stem-cell therapy is a promising method for treating patients with a wide range of diseases and injuries. Increasing government funding of scientific research has promoted rapid developments in stem-cell research in China, as evidenced by the substantial increase in the number and quality of publications in the past 5...

journal_title：Human gene therapy

authors： Hu L,Zhao B,Wang S

更新日期：2018-02-01 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：:Abstract Our studies have shown that coinjection of conventional single-stranded adeno-associated virus 2 (ssAAV2) vectors carrying the enhanced green fluorescent protein (EGFP) gene with self-complementary (sc) AAV2-T cell protein tyrosine phosphatase (TC-PTP) and scAAV2-protein phosphatase-5 (PP5) vectors resulted i...

journal_title：Human gene therapy

authors： Jayandharan GR,Zhong L,Sack BK,Rivers AE,Li M,Li B,Herzog RW,Srivastava A

更新日期：2010-03-01 00:00:00

abstract：:Twenty-eight patients with advanced neovascular age-related macular degeneration (AMD) were given a single intravitreous injection of an E1-, partial E3-, E4-deleted adenoviral vector expressing human pigment epithelium- derived factor (AdPEDF.11). Doses ranging from 10(6) to 10(9.5) particle units (PU) were investiga...

journal_title：Human gene therapy

authors： Campochiaro PA,Nguyen QD,Shah SM,Klein ML,Holz E,Frank RN,Saperstein DA,Gupta A,Stout JT,Macko J,DiBartolomeo R,Wei LL

更新日期：2006-02-01 00:00:00

abstract：:Herpes simplex virus thymidine kinase (HSV-tk) gene therapy for brain tumors depends on ganciclovir (GCV) and its transport across the blood-brain tumor barrier (BBTB). We examined whether RMP-7, the bradykinin analog and potent BBTB permeabilizer, could enhance the efficacy of GCV treatment of brain tumors by increas...

journal_title：Human gene therapy

authors： LeMay DR,Kittaka M,Gordon EM,Gray B,Stins MF,McComb JG,Jovanovic S,Tabrizi P,Weiss MH,Bartus R,Anderson WF,Zlokovic BV

更新日期：1998-05-01 00:00:00

abstract：:The transfer of a drug resistance gene into hematopoietic cells is an approach being investigated to overcome the problem of myelosuppression produced by anticancer drugs. Chemotherapeutic agents are often given in combination in order to increase their effectiveness. Consequently, there is an advantage in designing v...

journal_title：Human gene therapy

authors： Beauséjour CM,Le NL,Létourneau S,Cournoyer D,Momparler RL

更新日期：1998-11-20 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