Myoblast gene therapy in canine mucopolysaccharidosis. I: Abrogation by an immune response to alpha-L-iduronidase.

abstract：:Immune cells are involved in the pathogenesis of osteoarthritis (OA). CD4(+) T cells were activated during the onset of OA and induced macrophage inflammatory protein (MIP)-1γ expression and subsequent osteoclast formation. We evaluated the effects of local knockdown of MIP-1γ in a mouse OA model induced by anterior c...

journal_title：Human gene therapy

authors： Shen PC,Lu CS,Shiau AL,Lee CH,Jou IM,Hsieh JL

更新日期：2013-10-01 00:00:00

abstract：:The objective of this phase II investigation is to assess the safety and efficacy of a plasmid mediated approach to induce angiogenesis/arteriogenesis with the angiomatrix protein Del-1 (developmentally regulated endothelial locus 1), in subjects with intermittent claudication (IC) secondary to peripheral arterial dis...

journal_title：Human gene therapy

authors： Rajagopalan S,Olin JW,Young S,Erikson M,Grossman PM,Mendelsohn FO,Regensteiner JG,Hiatt WR,Annex BH

更新日期：2004-06-01 00:00:00

abstract：:Mucopolysaccharidosis type II (MPS II) is a neuropathic lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase (IDS), which leads to the accumulation of glycosaminoglycans (GAGs). We demonstrated that biochemical alterations in the brains of MPS II mice are not corrected by bone marrow transplantat...

journal_title：Human gene therapy

authors： Wakabayashi T,Shimada Y,Akiyama K,Higuchi T,Fukuda T,Kobayashi H,Eto Y,Ida H,Ohashi T

更新日期：2015-06-01 00:00:00

abstract：:Recombinant poxviruses expressing immunomodulatory molecules together with specific antigens represent powerful vaccines for cancer immunotherapy. Recently, we and others have demonstrated, in vitro and in vivo, that coexpression of CD80 and CD86 costimulatory molecules enhances the immunogenic capacity of a recombina...

journal_title：Human gene therapy

authors： Feder-Mengus C,Schultz-Thater E,Oertli D,Marti WR,Heberer M,Spagnoli GC,Zajac P

更新日期：2005-03-01 00:00:00

abstract：:Adenoviruses are attractive vectors for gene transfer into cardiac muscle. However, their promiscuous tissue tropism, which leads to an ectopic expression of the transgene, is a considerable limitation. To restrict expression to cardiomyocytes, we have constructed two recombinant adenoviruses (Ad-MLC2-250betagal and A...

journal_title：Human gene therapy

authors： Griscelli F,Gilardi-Hebenstreit P,Hanania N,Franz WM,Opolon P,Perricaudet M,Ragot T

更新日期：1998-09-01 00:00:00

abstract：:Glaucoma, a group of optic neuropathies, is the leading cause of irreversible blindness. Neuronal apoptosis in glaucoma is primarily associated with high intraocular pressure caused by chronically impaired outflow of aqueous humor through the trabecular meshwork, a reticulum of mitotically inactive endothelial-like ce...

journal_title：Human gene therapy

authors： Loewen N,Fautsch MP,Peretz M,Bahler CK,Cameron JD,Johnson DH,Poeschla EM

更新日期：2001-11-20 00:00:00

abstract：:Human serum is known to inactivate many retroviruses, including murine leukemia viruses (MLV). Exposure of vectors based on MLV to human serum components would presumably decrease the efficiency of gene transfer in vivo. Human serum also lyses xenogeneic cells, which would affect the survival of retroviral vector pack...

journal_title：Human gene therapy

authors： Russell DW,Berger MS,Miller AD

更新日期：1995-05-01 00:00:00

abstract：:The T cell co-stimulatory molecule B7-1 was transduced into a poorly immunogenic murine neuroblastoma cell line (Neuro-2a, N-2a) alone or in combination with MHC class II genes to test the ability of these genes to stimulate antitumor immunity. N-2a cells transduced with B7-1 exhibited reduced tumorigenicity, whereas ...

journal_title：Human gene therapy

authors： Heuer JG,Tucker-McClung C,Gonin R,Hock RA

更新日期：1996-11-10 00:00:00

abstract：:Administration of recombinant adenoviral (AdV) vectors to animals can lead to inflammatory and immune responses. For therapeutic indications in which repeated treatment is necessary, such as cystic fibrosis (CF), these responses can limit the therapeutic usefulness of the vector. In principle, the utility of the vecto...

journal_title：Human gene therapy

authors： Chu Q,St George JA,Lukason M,Cheng SH,Scheule RK,Eastman SJ

更新日期：2001-03-20 00:00:00

abstract：:At present there is no known effective pharmacological therapy for acute lung injury (ALI). Because keratinocyte growth factor (KGF) promotes epithelial cell growth, intratracheal administration of KGF has the possibility of restoring lung tissue integrity in injured lungs and improving patient outcomes. However, trea...

journal_title：Human gene therapy

authors： Baba Y,Yazawa T,Kanegae Y,Sakamoto S,Saito I,Morimura N,Goto T,Yamada Y,Kurahashi K

更新日期：2007-02-01 00:00:00

abstract：:Dystrophic epidermolysis bullosa (DEB) comprises a family of inherited blistering skin disorders for which no corrective therapy currently exists. In the most severe form, the Hallopeau-Siemens subtype (RDEB-HS), the epidermal adhesion protein collagen VII is absent from the skin as a consequence of null mutations in ...

journal_title：Human gene therapy

authors： Mecklenbeck S,Compton SH,Mejía JE,Cervini R,Hovnanian A,Bruckner-Tuderman L,Barrandon Y

更新日期：2002-09-01 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：: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：:DNA expression vectors may be administered to patients like conventional medicines to have a finite and controlled duration of action. The clinical application of these medicines will require a precise understanding of the kinetics of the administered gene, the mRNA transcript, and the gene product. The apparent kinet...

journal_title：Human gene therapy

authors： Ledley TS,Ledley FD

更新日期：1994-06-01 00:00:00

abstract：:Human bocavirus type-1 (HBoV1) has a high tropism for the apical membrane of human airway epithelia. The packaging of a recombinant adeno-associated virus 2 (rAAV2) genome into HBoV1 capsid produces a chimeric vector (rAAV2/HBoV1) that also efficiently transduces human airway epithelia. As such, this vector is attract...

journal_title：Human gene therapy

authors： Yan Z,Feng Z,Sun X,Zhang Y,Zou W,Wang Z,Jensen-Cody C,Liang B,Park SY,Qiu J,Engelhardt JF

更新日期：2017-08-01 00:00:00

abstract：:Systemic administration of adenoviral vectors leads to activation of innate and antigen-specific immunity. In an attempt to diminish T and B cell-specific immune responses to E1-deleted adenoviral vectors, capsid proteins were modified with various activated monomethoxypolyethylene glycols (MPEGs). The impact of this ...

journal_title：Human gene therapy

authors： Croyle MA,Chirmule N,Zhang Y,Wilson JM

更新日期：2002-10-10 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：: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：: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：:Niemann-Pick type C (NP-C) disease is a neurodegenerative disorder characterized neuropathologically by ballooned neurons distended with lipid storage and widespread neuronal loss. Neural stem cells (NSC) derived from NP-C disease models have decreased ability for self-renewal and neuronal differentiation. Investigati...

journal_title：Human gene therapy

authors： Lee H,Kang JE,Lee JK,Bae JS,Jin HK

更新日期：2013-07-01 00:00:00

abstract：:The first report of in vivo gene delivery to the retina dates back to 1987 when a retroviral vector was injected intraocularly in newborn mice. Later came the observation that retinal cells could be successfully transduced using adenoviral and then adeno-associated and lentiviral vectors. By 2000, it had become clear ...

journal_title：Human gene therapy

authors： Auricchio A,Smith AJ,Ali RR

更新日期：2017-11-01 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：: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：: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：:Deficiency of glycogen branching enzyme (GBE) causes glycogen storage disease type IV (GSD IV), which is characterized by the accumulation of a less branched, poorly soluble form of glycogen called polyglucosan (PG) in multiple tissues. This study evaluates the efficacy of gene therapy with an adeno-associated viral (...

journal_title：Human gene therapy

authors： Yi H,Zhang Q,Brooks ED,Yang C,Thurberg BL,Kishnani PS,Sun B

更新日期：2017-03-01 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：:At present, much more studies have focused on the role of microRNAs in osteoporosis, but the more specific role of microRNA-150-3p (miR-150-3p) in osteoporosis still needs full exploration. We aim at investigating the role of miR-150-3p in osteoporosis and at exploring the related mechanisms. Bone marrow mesenchymal s...

journal_title：Human gene therapy

authors： Qiu M,Zhai S,Fu Q,Liu D

更新日期：2021-01-22 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：:Deficiencies in different steps of purine metabolism give rise to a number of human inherited disorders. Lesch-Nyhan syndrome is a severe neurological disorder, caused by a deficiency in the purine salvage enzyme hypoxanthine phosphoribosyltransferase (HPRT). HPRT-deficient mice have been generated, but have proved to...

journal_title：Human gene therapy

authors： Redhead NJ,Selfridge J,Wu CL,Melton DW

更新日期：1996-08-20 00:00:00

abstract：:Umbilical cord blood (CB) from the early gestational human fetus is recognized as a rich source of hematopoietic stem cells. To examine the value of fetal CB for gene therapy of inborn immunohematopoietic disorders, we tested the feasibility of genetic modification of CD34(+) cells from CB at weeks 24 to 34 of pregnan...

journal_title：Human gene therapy

authors： Luther-Wyrsch A,Costello E,Thali M,Buetti E,Nissen C,Surbek D,Holzgreve W,Gratwohl A,Tichelli A,Wodnar-Filipowicz A

更新日期：2001-03-01 00:00:00