Correction: Recent development of AAV-based gene therapies for inner ear disorders.

abstract：:Platelet-derived growth factor-BB (PDGF-BB) stimulates repair of healing-impaired chronic wounds such as diabetic ulcers and periodontal lesions. However, limitations in predictability of tissue regeneration occur due, in part, to transient growth factor bioavailability in vivo. Here, we report that gene delivery of P...

journal_title：Gene therapy

authors： Chang PC,Seol YJ,Cirelli JA,Pellegrini G,Jin Q,Franco LM,Goldstein SA,Chandler LA,Sosnowski B,Giannobile WV

更新日期：2010-01-01 00:00:00

abstract：:The recent surge of DNA sequence information resulting from the efforts of agencies interested in deciphering the human genetic code has facilitated technological developments that have been critical in the identification of genes associated with numerous disease pathologies. In addition, these efforts have opened the...

journal_title：Gene therapy

authors： Gruenert DC,Novelli G,Dallapiccola B,Colosimo A

更新日期：2002-06-01 00:00:00

abstract：:The proteasome is a multisubunit cytosolic protein complex responsible for degrading cytosolic proteins. Several studies have implicated its involvement in the processing of viral particles used for gene delivery, thereby limiting the efficiency of gene transfer. Peptide-based nonviral gene delivery systems are suffic...

journal_title：Gene therapy

authors： Kim J,Chen CP,Rice KG

更新日期：2005-11-01 00:00:00

abstract：:Mucopolysaccharidosis type I (MPSI) is an autosomic recessive, lysosomal storage disorder due to the deficit of the enzyme α-L-iduronidase (IDUA). The disease accounts for a general impairment of tissue and organ functions, mainly including heart disease, corneal clouding, organomegaly, skeletal malformations and join...

journal_title：Gene therapy

authors： Piller Puicher E,Tomanin R,Salvalaio M,Friso A,Hortelano G,Marin O,Scarpa M

更新日期：2012-04-01 00:00:00

abstract：:Confinement of transgene expression to target cells is highly desirable in gene therapy. Current strategies of transcriptional targeting to tumors usually rely on tissue-specific promoters to control gene expression. However, such promoters generally have much lower activity than the constitutive viral promoters. We h...

journal_title：Gene therapy

authors： Fu X,Meng F,Tao L,Jin A,Zhang X

更新日期：2003-08-01 00:00:00

abstract：:Ideal methods for human gene therapy will eventually include direct gene transfer to defective tissues in a patient in vivo. Toward that goal, we have used high titer, pseudotyped retroviral vectors expressing genes for the Escherichia coli beta-galactosidase (lacZ) or hepatitis B virus surface antigen (HBsAg) to infe...

journal_title：Gene therapy

authors： Miyanohara A,Yee JK,Bouic K,LaPorte P,Friedmann T

更新日期：1995-03-01 00:00:00

abstract：:Advances in islet transplantation have encouraged efforts to create alternative insulin-secreting cells that overcome limitations associated with current therapies. We have recently demonstrated durable correction of murine and porcine diabetes by syngeneic and autologous implantation, respectively, of primary hepatoc...

journal_title：Gene therapy

authors： Chen NK,Tan SY,Udolph G,Kon OL

更新日期：2010-05-01 00:00:00

abstract：:Aptamers are oligonucleotides evolved in vitro or in nature to bind target ligands with high affinity and specificity. They are emerging as powerful tools in the fields of therapeutics, drug development, target validation and diagnostics. Aptamers are attractive alternatives to antibody- and small-molecule-based thera...

journal_title：Gene therapy

authors： Que-Gewirth NS,Sullenger BA

更新日期：2007-02-01 00:00:00

abstract：:Gene transfer vectors encoding two or more genes are potentially powerful research tools and are poised to play an increasingly important role in gene therapy applications. Common strategies employed to express more than one transgene per vector include the use of multiple promoters, internal ribosome entry site (IRES...

journal_title：Gene therapy

authors： Curtin JA,Dane AP,Swanson A,Alexander IE,Ginn SL

更新日期：2008-03-01 00:00:00

abstract：:To achieve high transgene expression in the liver, we have compared the reporter gene expression among various murine retroviral long terminal repeats (LTRs) or leader sequences in vitro. Transient reporter gene expression assays revealed the highest gene expression by the polycythemic strain of spleen focus-forming v...

journal_title：Gene therapy

authors： Ohnishi N,Itoh K,Itoh Y,Baum C,Higashitsuji H,Yamaguchi K,Tsuji T,Okanoue T,Fujita J

更新日期：2002-02-01 00:00:00

abstract：:T-cell dysfunction is thought to be central to the immunodeficiency state seen in patients with the Wiskott-Aldrich syndrome (WAS). Aspects of the WAS phenotype have been corrected in other cell types on introduction of the normal WAS protein (WASP), but the potential for correction of the T-cell defects has not been ...

journal_title：Gene therapy

authors： Strom TS,Gabbard W,Kelly PF,Cunningham JM,Nienhuis AW

更新日期：2003-05-01 00:00:00

abstract：:Recently, there has been an increasing level of interest in electroporation for gene delivery due to the site-specific nature of the delivery, as well as the high efficiency of the method. Electroporation involves the application of a pulsed electric field to cells to enhance cell permeability, resulting in the transi...

journal_title：Gene therapy

authors： Liu F,Huang L

更新日期：2002-08-01 00:00:00

abstract：:We explored the possibility of using a genetic approach to inhibit integrin-mediated endothelial cell adhesion and survival. We constructed recombinant adenoviruses (Ads) expressing chimeric proteins consisting of the cytoplasmic and transmembrane domains of integrin beta1 (CH1), beta3 (CH3) or the beta1 transmembrane...

journal_title：Gene therapy

authors： Oguey D,George PW,Rüegg C

更新日期：2000-08-01 00:00:00

abstract：:We have compared the efficacy of daily injection of recombinant leptin protein (rh-leptin) with adenovirus-mediated delivery of the murine or human leptin gene (Ad-leptin) for treatment of obesity in the obese (ob/ob) mouse model. We demonstrate an improved correction profile for obesity and associated surrogate marke...

journal_title：Gene therapy

authors： Morsy MA,Gu MC,Zhao JZ,Holder DJ,Rogers IT,Pouch WJ,Motzel SL,Klein HJ,Gupta SK,Liang X,Tota MR,Rosenblum CI,Caskey CT

更新日期：1998-01-01 00:00:00

abstract：:Genetic modification of tumor cells with the gene for the B7.1 or with the genes for cytokines results in increased tumor cell immunogenicity. In the work reported here, immunization of naive animals with either B7.1 or gamma-IFN gene-modified MCA106 tumor cells effectively protects the host from subsequent challenge ...

journal_title：Gene therapy

authors： Yang S,Vervaert CE,Seigler HF,Darrow TL

更新日期：1999-02-01 00:00:00

abstract：:For certain gene therapy applications, the simultaneous delivery of multiple genes would allow for novel therapies. In the case of adeno-associated virus (AAV) vectors, the limited packaging capacity greatly restricts current methods of carrying multiple transgene cassettes. To address this issue, a recombinant AAV (r...

journal_title：Gene therapy

authors： Foti SB,Samulski RJ,McCown TJ

更新日期：2009-11-01 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：:We have shown that various forms of oligonucleotides, chimeric RNA-DNA oligonucleotide (RDO) and single-stranded oligodeoxynucleotide (ODN), are capable of chromosomal gene alterations in mammalian cells. Using two ODNs we corrected an inactivating mutation in the tyrosinase gene and introduced an activating mutation ...

journal_title：Gene therapy

authors： Alexeev V,Igoucheva O,Yoon K

更新日期：2002-12-01 00:00:00

abstract：:Current generation adenovirus (Ad) vectors are deleted for the E1 region of genes and require propagation in E1 expressing 293 cells. Expression of genes delivered by Ad vectors into immunocompetent hosts is generally transient since the current vectors are not completely replication defective. Viral proteins expresse...

journal_title：Gene therapy

authors： Amalfitano A,Chamberlain JS

更新日期：1997-03-01 00:00:00

abstract：:By-pass surgery and percutaneous transluminal (coronary) angioplasty, PT(C)A, are standard techniques for the treatment of vascular occlusions. Their usefulness is limited by by-pass graft failure and restenosis occurring after the procedures. Twenty percent of patients treated with PTCA/PTA need a new revascularizati...

journal_title：Gene therapy

authors： Rutanen J,Puhakka H,Ylä-Herttuala S

更新日期：2002-11-01 00:00:00

abstract：:Lentivirus can be engineered to be a highly potent vector for gene therapy applications. However, generation of clinical grade vectors in enough quantities for therapeutic use is still troublesome and limits the preclinical and clinical experiments. As a first step to solve this unmet need we recently introduced a bac...

journal_title：Gene therapy

authors： Lesch HP,Laitinen A,Peixoto C,Vicente T,Makkonen KE,Laitinen L,Pikkarainen JT,Samaranayake H,Alves PM,Carrondo MJ,Ylä-Herttuala S,Airenne KJ

更新日期：2011-06-01 00:00:00

abstract：:Active disease of tuberculosis (TB) can be developed decades later by either a relapse of the initial infection (endogenous reactivation) or by an entrance of the secondary infection (exogenous reinfection), since the current chemotherapy cannot lead to complete elimination of tuberculosis. Although the immunotherapeu...

journal_title：Gene therapy

authors： Ha SJ,Jeon BY,Youn JI,Kim SC,Cho SN,Sung YC

更新日期：2005-04-01 00:00:00

abstract：:The corticospinal tract (CST) is extensively used as a model system for assessing potential therapies to enhance neuronal regeneration and functional recovery following spinal cord injury (SCI). However, efficient transduction of the CST is challenging and remains to be optimised. Recombinant adeno-associated viral (A...

journal_title：Gene therapy

authors： Hutson TH,Verhaagen J,Yáñez-Muñoz RJ,Moon LD

更新日期：2012-01-01 00:00:00

abstract：:Parkinson's disease is a prevalent progressive degenerative disorder of the elderly. There is a current need for novel therapeutic strategies because the standard levodopa pharmacotherapy is only temporarily efficacious. Recently, there have been some high-profile successful preclinical results obtained in animal mode...

journal_title：Gene therapy

authors： Manfredsson FP,Lewin AS,Mandel RJ

更新日期：2006-03-01 00:00:00

abstract：:A fully intact immune system would be expected to hinder the efficacy of oncolytic virotherapy by inhibiting viral replication. Simultaneously, however, it may also enhance antitumor therapy through initiation of proinflammatory, antiviral cytokine responses at the tumor site. The aim of this study was to investigate ...

journal_title：Gene therapy

authors： Galivo F,Diaz RM,Wongthida P,Thompson J,Kottke T,Barber G,Melcher A,Vile R

更新日期：2010-02-01 00:00:00

abstract：:X-linked chronic granulomatous disease (X-CGD) is an inherited immunodeficiency with absent phagocyte NADPH-oxidase activity caused by defects in the gene-encoding gp91(phox). Here, we evaluated strategies for less intensive conditioning for gene therapy of genetic blood disorders without selective advantage for gene ...

journal_title：Gene therapy

authors： Sadat MA,Dirscherl S,Sastry L,Dantzer J,Pech N,Griffin S,Hawkins T,Zhao Y,Barese CN,Cross S,Orazi A,An C,Goebel WS,Yoder MC,Li X,Grez M,Cornetta K,Mooney SD,Dinauer MC

更新日期：2009-12-01 00:00:00

abstract：:To evaluate the use of HSV-based vectors for arthritis gene therapy we have constructed a first-generation, ICP4 deficient, replication defective herpes simplex virus (HSV) vector (S/0-) and a second-generation HSV vector derivative (T/0-) deficient for the immediate-early genes ICP4, 22 and 27, each carrying a solubl...

journal_title：Gene therapy

authors： Oligino T,Ghivizzani S,Wolfe D,Lechman E,Krisky D,Mi Z,Evans C,Robbins P,Glorioso J

更新日期：1999-10-01 00:00:00

abstract：:We have comparatively evaluated the efficiency of a series of retroviral vectors transducing the gp91-phox gene, whose defects are responsible for impaired production of superoxide anion (O2-) by phagocytic cells and lead to the X-linked form of chronic granulomatous disease (X-CGD). These vectors included four constr...

journal_title：Gene therapy

authors： Zentilin L,Qin G,Tafuro S,Dinauer MC,Baum C,Giacca M

更新日期：2000-01-01 00:00:00

abstract：:Efficient gene delivery is a critical obstacle for gene therapy that must be overcome. Until current limits of gene delivery technology are solved, identification of systems with bystander effects is highly desirable. As an anticancer agent, radioactive iodine (131)I has minimal toxicity. The physical characteristics ...

journal_title：Gene therapy

authors： Mitrofanova E,Unfer R,Vahanian N,Link C

更新日期：2006-07-01 00:00:00

abstract：:Gene delivery of angiogenic growth factors is a promising approach for the treatment of ischemic cardiovascular diseases. However, success of this new therapeutic principle is hindered by the lack of critical understanding as to how disease pathology affects the efficiency of gene delivery and/or the downstream signal...

journal_title：Gene therapy

authors： Qian HS,Liu P,Huw LY,Orme A,Halks-Miller M,Hill SM,Jin F,Kretschmer P,Blasko E,Cashion L,Szymanski P,Vergona R,Harkins R,Yu J,Sessa WC,Dole WP,Rubanyi GM,Kauser K

更新日期：2006-09-01 00:00:00