abstract：:Evidence has documented the tumor-promoting properties of long non-coding RNA (lncRNA) FOXD2 adjacent opposite strand RNA 1 (FOXD2-AS1) in many cancers. However, little is known about its role in gallbladder cancer. Here, we aimed to characterize the functional relevance of lncRNA FOXD2-AS1 in gallbladder cancer and t...

journal_title：Gene therapy

authors： Gao J,Dai C,Yu X,Yin XB,Liao WJ,Huang Y,Zhou F

更新日期：2020-09-11 00:00:00

abstract：:Adeno-associated virus (AAV)-mediated gene delivery systems have been shown to be effective tools for gene manipulation in the inner ear. For example, hair cells (HCs) and multiple other cell types can be transduced by the local injection of AAVs into the inner ear. However, application of the AAV-mediated CRISPR/Cas9...

journal_title：Gene therapy

authors： Kang W,Zhao X,Sun Z,Dong T,Jin C,Tong L,Zhu W,Tao Y,Wu H

更新日期：2020-08-01 00:00:00

abstract：:An amendment to this paper has been published and can be accessed via a link at the top of the paper. ...

journal_title：Gene therapy

authors： Lan Y,Tao Y,Wang Y,Ke J,Yang Q,Liu X,Su B,Wu Y,Lin CP,Zhong G

更新日期：2020-08-01 00:00:00

abstract：:The GM2-gangliosidoses are neurological diseases causing premature death, thus developing effective treatment protocols is urgent. GM2-gangliosidoses result from deficiency of a lysosomal enzyme β-hexosaminidase (Hex) and subsequent accumulation of GM2 gangliosides. Genetic changes in HEXA, encoding the Hex α subunit,...

journal_title：Gene therapy

authors： Ou L,Przybilla MJ,Tăbăran AF,Overn P,O'Sullivan MG,Jiang X,Sidhu R,Kell PJ,Ory DS,Whitley CB

更新日期：2020-05-01 00:00:00

abstract：:For patients with metastatic prostate cancer, the 5-year survival rate of 31% points to a need for novel therapies and improvement of existing modalities. We propose that p53 gene therapy and chemotherapy, when combined, will provide superior tumor cell killing for the treatment of prostate carcinoma. To this end, we ...

journal_title：Gene therapy

authors： Tamura RE,Lana MG,Costanzi-Strauss E,Strauss BE

更新日期：2020-02-01 00:00:00

abstract：:Cancer therapies that target a single protein or pathway may be limited by their specificity, thus missing key players that control cellular proliferation and contributing to the failure of the treatment. We propose that approaches to cancer therapy that hit multiple targets would limit the chances of escape. To this ...

journal_title：Gene therapy

authors： Xande JG,Dias AP,Tamura RE,Cruz MC,Brito B,Ferreira RA,Strauss BE,Costanzi-Strauss E

更新日期：2020-02-01 00:00:00

abstract：:Studies reviewed show that lentiviral gene therapy directed either at inhibiting the synthesis of brain acetaldehyde generated from ethanol or at degrading brain acetaldehyde fully prevent ethanol intake by rats bred for their high alcohol preference. However, after animals have chronically consumed alcohol, the above...

journal_title：Gene therapy

authors： Yedy Israel,Quintanilla ME,Ezquer F,Morales P,Rivera-Meza M,Karahanian E,Ezquer M,Herrera-Marschitz M

更新日期：2019-11-01 00:00:00

abstract：:Self-amplifying RNA (saRNA) is a promising biotherapeutic tool that has been used as a vaccine against both infectious diseases and cancer. saRNA has been shown to induce protein expression for up to 60 days and elicit immune responses with lower dosing than messenger RNA (mRNA). Because saRNA is a large (~9500 nt), n...

journal_title：Gene therapy

authors： Blakney AK,McKay PF,Yus BI,Aldon Y,Shattock RJ

更新日期：2019-09-01 00:00:00

abstract：:Excessive activated T-cell proliferation was observed in vivo in one patient after an anti-CD19-chimeric antigen receptor (CAR) T-cell infusion. The patient, who had chemotherapy refractory and CD19+ diffuse large B-cell lymphoma (DLBCL), received an anti-CD19 CAR T-cell infusion following conditioning chemotherapy (f...

journal_title：Gene therapy

authors： Zhang WY,Liu Y,Wang Y,Nie J,Guo YL,Wang CM,Dai HR,Yang QM,Wu ZQ,Han WD

更新日期：2018-06-01 00:00:00

abstract：:Intervertebral disc degeneration (IVDD) is a multi-factorial process characterized by phenotypic and genotypic changes, which leads to low back pain and disability. Prolonged imbalance between anabolism and catabolism in discs alters their composition resulting in progressive loss of proteoglycans and hydration leadin...

journal_title：Gene therapy

authors： Sampara P,Banala RR,Vemuri SK,Av GR,Gpv S

更新日期：2018-04-01 00:00:00

abstract：:Until recently, adeno-associated virus 9 (AAV9) was considered the AAV serotype most effective in crossing the blood-brain barrier (BBB) and transducing cells of the central nervous system (CNS), following systemic injection. However, a newly engineered capsid, AAV-PHP.B, is reported to cross the BBB at even higher ef...

journal_title：Gene therapy

authors： Rincon MY,de Vin F,Duqué SI,Fripont S,Castaldo SA,Bouhuijzen-Wenger J,Holt MG

更新日期：2018-04-01 00:00:00

abstract：:Lentiviral vectors pseudotyped with the baculovirus envelope protein GP64 transduce primary cultures of human airway epithelia (HAE) at their apical surface. Our goal in this study was to harness a directed evolution approach to develop a novel envelope glycoprotein with increased transduction properties for HAE. Usin...

journal_title：Gene therapy

authors： Sinn PL,Hwang BY,Li N,Ortiz JLS,Shirazi E,Parekh KR,Cooney AL,Schaffer DV,McCray PB Jr

更新日期：2017-10-01 00:00:00

abstract：:Despite significant advances in basic research, the treatment of degenerative diseases of the nervous system remains one of the greatest challenges for translational medicine. The childhood onset motor neuron disorder spinal muscular atrophy (SMA) has been viewed as one of the more tractable targets for molecular ther...

journal_title：Gene therapy

authors： Talbot K,Tizzano EF

更新日期：2017-09-01 00:00:00

abstract：:The production of high-titer recombinant adeno-associated virus (rAAV) vector is essential for treatment of genetic diseases affecting the retina and choroid, where anatomical constraints may limit injectable volumes. Problematically, cytotoxicity arising from overexpression of the transgene during vector production f...

journal_title：Gene therapy

authors： Reid CA,Boye SL,Hauswirth WW,Lipinski DM

更新日期：2017-08-01 00:00:00

abstract：:Transplantation of allogeneic cells as well as of genetically corrected autologous cells are potent approaches to restore cellular functions in patients suffering from genetic diseases. The recipient's immune responses against non-self-antigens may compromise the survival of the grafted cells. Recipients of the graft ...

journal_title：Gene therapy

authors： Bots STF,Hoeben RC

更新日期：2017-07-01 00:00:00

abstract：:Adeno-associated virus (AAV) gene therapy constitutes a powerful tool for the treatment of neurodegenerative diseases. While AAVs are generally administered systemically to newborns in preclinical studies of neurological disorders, in adults the maturity of the blood-brain barrier (BBB) must be considered when selecti...

journal_title：Gene therapy

authors： Bey K,Ciron C,Dubreil L,Deniaud J,Ledevin M,Cristini J,Blouin V,Aubourg P,Colle MA

更新日期：2017-05-01 00:00:00

abstract：:Morphine appears to be the most active metabolite of heroin; therefore, the effects of morphine are important in understanding the ramifications of heroin abuse. Opioid physical dependence (withdrawal response) may have very long-lasting effects on the motivation for reward, including the incubation of cue-induced dru...

journal_title：Gene therapy

authors： Iida T,Yi H,Liu S,Ikegami D,Zheng W,Liu Q,Takahashi K,Kashiwagi Y,Goins WF,Glorioso JC,Hao S

更新日期：2017-05-01 00:00:00

abstract：:Recombinant human platelet-derived growth factor-BB (rhPDGF-BB) promotes soft tissue and bone healing, and is Food and Drug Administration-approved for treatment of diabetic ulcers and periodontal defects. The short half-life of topical rhPDGF-BB protein application necessitates bolus, high-dose delivery. Gene therapy...

journal_title：Gene therapy

authors： Plonka AB,Khorsand B,Yu N,Sugai JV,Salem AK,Giannobile WV,Elangovan S

更新日期：2017-01-01 00:00:00

abstract：:Breast cancer is the leading cancer diagnosed in women and the second leading cause of cancer-related deaths in women. Current limitations to standard chemotherapy in the clinic are extensively researched, including problems arising from repeated treatments with the same drugs. The phenomenon that cancer cells become ...

journal_title：Gene therapy

authors： Jones SK,Merkel OM

更新日期：2016-12-01 00:00:00

abstract：:The ability to restrict gene delivery and expression to particular cell types is of paramount importance for many types of gene therapy, especially in the lung. The alveolar epithelial type I (ATI) cell, in particular, is an attractive cell type to target, as it comprises 95% of the internal surface area of the lung. ...

journal_title：Gene therapy

authors： Gottfried L,Lin X,Barravecchia M,Dean DA

更新日期：2016-10-01 00:00:00

abstract：:Adoptive cell therapy with chimeric antigen receptor (CAR)-modified T cells showed remarkable therapeutic efficacy in the treatment of leukaemia/lymphoma. However, the application to a variety of cancer entities is often constricted by the non-availability of a single chain antibody (scFv), which is usually the target...

journal_title：Gene therapy

authors： Faitschuk E,Nagy V,Hombach AA,Abken H

更新日期：2016-10-01 00:00:00

abstract：:The canine is the most important large animal model for testing novel hemophilia A (HA) treatment. It is often necessary to use canine factor VIII (cFIII) gene or protein for the evaluation of HA treatment in the canine model. However, different biological properties between cFVIII and human FVIII (hFVIII) indicated t...

journal_title：Gene therapy

authors： Wang Q,Dong B,Firrman J,Wu W,Roberts S,Moore AR,Liu LS,Chin MP,Diao Y,Kost J,Xiao W

更新日期：2016-07-01 00:00:00

abstract：:Intravenous delivery of adenoviruses is the optimal route for many gene therapy applications. Once in the blood, coagulation factor X (FX) binds to the adenovirus capsid and protects the virion from natural antibody and classical complement-mediated neutralisation in mice. However, to date, no studies have examined th...

journal_title：Gene therapy

authors： Duffy MR,Doszpoly A,Turner G,Nicklin SA,Baker AH

更新日期：2016-07-01 00:00:00

abstract：:Acute lung injury (ALI) and acute respiratory distress syndrome (ARDS) are associated with diverse disorders and characterized by disruption of the alveolar-capillary barrier, leakage of edema fluid into the lung, and substantial inflammation leading to acute respiratory failure. Gene therapy is a potentially powerful...

journal_title：Gene therapy

authors： Lin X,Barravecchia M,Kothari P,Young JL,Dean DA

更新日期：2016-06-01 00:00:00

abstract：:Current tools for the inhibition of microRNA (miR) function are limited to modified antisense oligonucleotides, sponges and decoy RNA molecules and none have been used to understand miR function during development. CRISPR/Cas-mediated deletion of miR sequences within the genome requires multiple chromosomal deletions ...

journal_title：Gene therapy

authors： Cao H,Yu W,Li X,Wang J,Gao S,Holton NE,Eliason S,Sharp T,Amendt BA

更新日期：2016-06-01 00:00:00

abstract：:Urothelial bladder cancer is the most common malignancy of the urinary tract. Although most cases are initially diagnosed as non-muscle-invasive, more than 80% of patients will develop recurrent or metastatic tumors. No effective therapy exists currently for late-stage metastatic tumors. By intravesical application, l...

journal_title：Gene therapy

authors： Zhang KX,Matsui Y,Lee C,Osamu O,Skinner L,Wang J,So A,Rennie PS,Jia WW

更新日期：2016-05-01 00:00:00

abstract：:Prostate cancer is the most common cancer in men of the western world. To date, no effective treatment exists for metastatic prostate cancer and consequently, there is an urgent need to develop new and improved therapeutics. In recent years, the therapeutic potential of RNA interference (RNAi) has been extensively exp...

journal_title：Gene therapy

authors： Evans JC,McCarthy J,Torres-Fuentes C,Cryan JF,Ogier J,Darcy R,Watson RW,O'Driscoll CM

更新日期：2015-10-01 00:00:00

abstract：:Schwann cells (SCs) in an injured peripheral nerve form pathways for regenerating axons. Although these cells initially support regeneration, SCs lose their pro-regenerative properties following a prolonged period of denervation. Gene transfer to SC can enhance their therapeutic potential. In this article, we compared...

journal_title：Gene therapy

authors： Hoyng SA,De Winter F,Gnavi S,van Egmond L,Attwell CL,Tannemaat MR,Verhaagen J,Malessy MJ

更新日期：2015-10-01 00:00:00

abstract：:In 2012, we reported that 5 out of 11 subjects in a clinical trial (NCT00372320) administering AdhAQP1 to radiation-damaged parotid glands showed increased saliva flow rates and decreased symptoms over the initial 42 days. AdhAQP1 is a first-generation, E1-deleted, replication-defective, serotype 5 adenoviral vector e...

journal_title：Gene therapy

authors： Zheng C,Baum BJ,Liu X,Goldsmith CM,Perez P,Jang SI,Cotrim AP,McCullagh L,Ambudkar IS,Alevizos I

更新日期：2015-09-01 00:00:00

abstract：:Chimeric antigen receptor (CAR) T-cell therapies have demonstrated durable and potentially curative therapeutic efficacy against B-cell leukemia in clinical trials. A CAR strategy can target any tumor surface antigens as long as an antigen-binding receptor can be generated. New CARs that target solid tumors and have t...

journal_title：Gene therapy

authors： Wu MR,Zhang T,DeMars LR,Sentman CL

更新日期：2015-08-01 00:00:00

abstract：:Gene therapy with Plasmid AMEP (antiangiogenic metargidin peptide) has recently been studied as a potential targeted therapy for melanoma. This plasmid is designed to downregulate α5β1 and αvβ3 integrins. In our study, electroporation was used as a nonviral delivery system. We investigated the antiangiogenic and direc...

journal_title：Gene therapy

authors： Bosnjak M,Dolinsek T,Cemazar M,Kranjc S,Blagus T,Markelc B,Stimac M,Zavrsnik J,Kamensek U,Heller L,Bouquet C,Turk B,Sersa G

更新日期：2015-07-01 00:00:00

abstract：:Foamy virus (FV) vectors are promising for hematopoietic stem cell (HSC) gene therapy but preclinical data on the clonal composition of FV vector-transduced human repopulating cells is needed. Human CD34(+) human cord blood cells were transduced with an FV vector encoding a methylguanine methyltransferase (MGMT)P140K ...

journal_title：Gene therapy

authors： Olszko ME,Adair JE,Linde I,Rae DT,Trobridge P,Hocum JD,Rawlings DJ,Kiem HP,Trobridge GD

更新日期：2015-07-01 00:00:00

abstract：:Cellular immunity against cancer can be achieved with viral vector- and DNA-based immunizations. In preclinical studies, cancer vaccines are very potent, but in clinical trials these potencies are not achieved yet. Thus, a rational approach to improve cancer vaccines is warranted. We previously demonstrated that the r...

journal_title：Gene therapy

authors： Ip PP,Boerma A,Walczak M,Oosterhuis K,Haanen JB,Schumacher TN,Nijman HW,Daemen T

更新日期：2015-07-01 00:00:00

abstract：:We created Na(+)/HCO3(-) cotransporter 1 (NBCe1) p.W516* knock-in mice as a model of isolated proximal renal tubular acidosis showing early lethality associated with severe metabolic acidosis to investigate the therapeutic effects of prenatal alkalization or posttranscriptional control 124 (PTC124). NBCe1(W516*/W516*)...

journal_title：Gene therapy

authors： Fang YW,Yang SS,Chau T,Nakamura M,Yamazaki O,Seki G,Yamada H,Hsu HM,Cheng CJ,Lin SH

更新日期：2015-05-01 00:00:00

abstract：:The current therapies to treat hepatitis B virus (HBV) infection are limited. Recently, clustered regularly interspaced short palindromic repeat (CRISPR) systems, originally identified in bacteria and archaea, have been found to consist of an RNA-based adaptive immune system that degrades complimentary sequences of in...

journal_title：Gene therapy

authors： Zhen S,Hua L,Liu YH,Gao LC,Fu J,Wan DY,Dong LH,Song HF,Gao X

更新日期：2015-05-01 00:00:00

abstract：:Intracerebral administration of recombinant adeno-associated vector (AAV) has been performed in several clinical trials. However, delivery into the brain requires multiple injections and is not efficient to target the spinal cord, thus limiting its applications. To assess widespread and less invasive strategies, we te...

journal_title：Gene therapy

authors： Hordeaux J,Dubreil L,Deniaud J,Iacobelli F,Moreau S,Ledevin M,Le Guiner C,Blouin V,Le Duff J,Mendes-Madeira A,Rolling F,Cherel Y,Moullier P,Colle MA

更新日期：2015-04-01 00:00:00

abstract：:Chronic infection with hepatitis B virus (HBV) puts individuals at high risk for complicating cirrhosis and liver cancer, but available treatment to counter the virus rarely eliminates infection. Although harnessing RNA interference (RNAi) to silence HBV genes has shown the potential, achieving efficient and durable s...

journal_title：Gene therapy

authors： Ivacik D,Ely A,Ferry N,Arbuthnot P

更新日期：2015-02-01 00:00:00

abstract：:Hyperammonemia is less severe in arginase 1 deficiency compared with other urea cycle defects. Affected patients manifest hyperargininemia and infrequent episodes of hyperammonemia. Patients typically suffer from neurological impairment with cortical and pyramidal tract deterioration, spasticity, loss of ambulation, s...

journal_title：Gene therapy

authors： Hu C,Tai DS,Park H,Cantero G,Cantero-Nieto G,Chan E,Yudkoff M,Cederbaum SD,Lipshutz GS

更新日期：2015-02-01 00:00:00

abstract：:The mouse is widely used as a model for DNA therapy and vaccination even though the efficiency of DNA delivery in higher mammals and humans is much less. The human APOBEC3 (A3) enzymes impact viral genomes by cytidine deamination, which introduces multiple uridine residues into single-stranded DNA, a process known as ...

journal_title：Gene therapy

authors： Kostrzak A,Henry M,Demoyen PL,Wain-Hobson S,Vartanian JP

更新日期：2015-01-01 00:00:00

abstract：:Mucopolysaccharidosis type IIIA (MPS-IIIA) or Sanfilippo A syndrome is a lysosomal storage genetic disease that results from the deficiency of the N-sulfoglucosamine sulfohydrolase (SGSH) protein, a sulfamidase required for the degradation of heparan sulfate glycosaminoglycans (GAGs). The accumulation of these macromo...

journal_title：Gene therapy

authors： Quiviger M,Arfi A,Mansard D,Delacotte L,Pastor M,Scherman D,Marie C

更新日期：2014-12-01 00:00:00