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Human Gene Therapy

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https://www.readbyqxmd.com/read/28530155/an-oncolytic-adenovirus-encoding-decorin-and-gm-csf-inhibits-tumor-growth-in-a-colorectal-tumor-model-by-targeting-pro-tumorigenic-signals-and-via-immune-activation
#1
Zhao Liu, Yuefeng Yang, Xiaoyan Zhang, Hao Wang, Weidong Xu, Hua Wang, FengJun Xiao, Zhigang Bai, Hongwei Yao, Xuemei Ma, Lan Jin, Chu-Tse Wu, Prem Seth, Zhongtao Zhang, Lisheng Wang
In advanced and metastatic stages of colorectal cancer (CRC), reduced sensitivity to conventional strategies is still a major obstacle to successful treatments. Decorin is an important regulator in the development and progression of various cancers. To examine if CRC patients have altered decorin levels, expression of decorin and its target genes, Met and vascular endothelial growth factor A (VEGFA) were analyzed in their tumors. Compared to normal tissues, decorin expression was reduced in CRC patients' tumors, while, there were increased Met and VEGFA levels...
May 20, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28530135/prolonged-expression-of-secreted-enzymes-in-dogs-after-liver-directed-delivery-of-i-sleeping-beauty-i-transposons-implications-for-non-viral-gene-therapy-of-systemic-disease
#2
Elena L Aronovich, Kendra Anne Hyland, Bryan C Hall, Jason B Bell, Erik R Olson, Myra Urness Rusten, David W Hunter, N Matthew Ellinwood, R Scott McIvor, Perry B Hackett
The non-viral, integrating Sleeping Beauty (SB) transposon system is efficient in treating systemic monogenic disease in mice including hemophilias A and B caused by deficiency of blood clotting factors and mucopolysaccharidosis (MPS) types I and VII caused by α-L-iduronidase (IDUA) and β-glucuronidase (GUSB) deficiency, respectively. We recently reported modified approaches of the hydrodynamics-based procedure to deliver transposons to the liver in dogs. Using the transgenic canine reporter secreted alkaline phosphatase (cSEAP), we demonstrated transgenic protein in the plasma for up to six weeks post-infusion...
May 20, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28530128/in-vivo-endocrine-secretion-of-prostacyclin-following-expression-of-a-cox-1-pgis-fusion-protein-in-the-salivary-glands-of-rats-via-non-viral-gene-therapy
#3
Zhimin Wang, Raymond L Benza, Lee Zourelias, Angela Sanguino, Ramaz Geguchadze, Kelly J Shields, Changgong Wu, Kristin B Highland, Michael J Passineau
Pulmonary arterial hypertension is a progressive disease that culminates in right heart failure and death. Prostacyclin(PGI2) and its derivatives are effective treatments for PAH when administered as continuous parenteral infusions. This treatment paradigm requires medical sophistication, and patients are at risk for complications from an indewelling catheter; drug interruptions may result in rebound pulmonary hypertension and death. We hypothesized that the salivary gland can be repurposed into an endogenous production site for circulating PGI2 through the expression of a fusion protein embodying cyclooxygenase-1(Cox1) and prostacyclin synthase(PGIS) domains...
May 20, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28530127/an-oncolytic-adenovirus-expressing-snord44-and-gas5-exhibits-anti-tumor-effect-in-colorectal-cancer-cells
#4
Sujing Yuan, Yu Wu, Yigang Wang, Jianhua Chen, Liang Chu
SNORD44 is a C/D box small nucleolar RNA, and low expresses in breast cancer and head and neck squamous cell carcinoma tissues. Its host gene is growth arrest specific transcript 5 (GAS5), which is a long noncoding RNA. GAS5 is down-regulated in colorectal cancer (CRC), and overexpression of GAS5 suppresses cell proliferation. However, the function of SNORD44 in CRC remains largely unknown, and the application of SNORD44 combined with GAS5 in CRC treatment has not been reported. In this study, the expression levels of SNORD44 and GAS5 were measured in CRC tissues by qRT-PCR...
May 20, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28510482/rod-outer-segment-development-influences-aav-mediated-photoreceptor-transduction-after-subretinal-injection
#5
Lolita Petit, Shan Ma, Shun-Yun Cheng, Guangping Gao, Claudio Punzo
Vectors based on the adeno-associated virus (AAV) are currently the preferred tools for delivering genes to photoreceptors (PR) in small and large animals. AAVs have been applied successfully in various models of PR dystrophies. However, unknown barriers still limit AAV's efficient application in several forms of severe PR degenerations, due to insufficient transgene expression and/or treated cells at the time of injection. Optimizations of PR gene therapy strategies will likely benefit from the identification of the cellular factors that influence PR transduction...
May 16, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28490211/aav-vectors-and-stem-cells-friends-or-foes
#6
Nolan Brown, Liujiang Song, Nageswara Rao Kollu, Matt Hirsch
The infusion of healthy stem cells into a patient, termed stem cell therapy, has shown great promise for the treatment of genetic and non-genetic diseases including mucopolysaccharidosis type 1, Parkinson's disease, multiple sclerosis, numerous immunodeficiency disorders, and aplastic anemia. Stem cells for cell therapy can be collected from the patient (autologous) or collected from another "healthy" individual (allogeneic). The use of allogenic stem cells is accompanied with the potentially fatal risk that the transplanted donor T cells will reject the patient's cells, a process termed graft-versus-host disease...
May 10, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28490200/human-bocavirus-type-1-capsid-facilitates-the-transduction-of-ferret-airways-by-adeno-associated-virus-genomes
#7
Ziying Yan, Zehua Feng, Xingshen Sun, Yulong Zhang, Wei Zou, Zekun Wang, Chandler Jensen-Cody, Bo Liang, Soo-Yeun Park, Jianming Qiu, John F Engelhardt
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 attractive for use in gene therapies to treat lung diseases such as cystic fibrosis. However, preclinical development of rAAV2/HBoV1 vectors has been hindered by the fact that humans are the only known host for HBoV1 infection...
May 10, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28478735/gene-therapy-of-adult-neuronal-ceroid-lipofuscinoses-with-crispr-cas9-in-zebrafish
#8
Xiaomin Yao, Xiaowei Liu, Yaguang Zhang, Yuhao Li, Chengjian Zhao, Shaohua Yao, Yu-Quan Wei
Adult-Onset Neuronal Ceroid Lipofuscinosis (ANCL), one of the neuronal ceroid lipofuscinosis (NCLs), is an inherited neurodegenerative disorder with progressive neuronal dysfunction. Recently, mutations in DNAJC5 gene that encodes Cysteine-String protein Alpha (CSPα) have been reported to be associated with familial Autosomal-Dominant ANCL (AD-ANCL). Here, we constructed an ANCL transgenic zebrafish model expressing human mutant DNAJC5 (mDNAJC5) gene under the control of a zebrafish neuron-specific promoter...
May 6, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28478695/prevention-of-neurocognitive-deficiency-in-mucopolysaccharidosis-type-ii-mice-by-cns-directed-aav9-mediated-iduronate-sulfatase-gene-transfer
#9
Kanut Laoharawee, Kelly M Podetz-Pedersen, Tam T Nguyen, Laura B Evenstar, Kelley F Kitto, Zhenhong Nan, Carolyn A Fairbanks, Walter C Low, Karen F Kozarsky, R Scott McIvor
Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is a rare X-linked recessive lysosomal disorder caused by defective Iduronate-2-sulfatase (IDS) resulting in accumulation of heparan sulfate and dermatan sulfate glycosaminoglycans (GAGs). Enzyme replacement is the only FDA-approved therapy available for MPS II, but it is expensive and does not improve neurologic outcomes in MPS II patients. We conducted this study to evaluate the effectiveness of adeno-associated virus (AAV) vector encoding human IDS delivered intracerebroventricularly in a murine model of MPS II...
May 6, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28478688/gene-delivery-of-activated-factor-vii-using-alternative-aav-serotype-improves-hemostasis-in-hemophiliac-mice-with-fviii-inhibitors-and-aav-neutralizing-antibodies
#10
Junjiang Sun, Baolai Hua, R Jude Samulski, Chengwen Li
While therapeutic expression of coagulation factors from adeno-associated virus (AAV) vectors has been successfully achieved in patients with hemophilia, neutralizing antibodies to the vector and inhibitory antibodies to the transgene severely limit efficacy. Indeed, approximately 40% of mice transduced with human Factor VIII using the AAV8 serotype developed inhibitory antibodies to Factor VIII (FVIII inhibitor), as well as extremely high titers (≥ 1:500) of neutralizing antibodies to AAV8. To correct hemophilia in these mice we used AAV9, a serotype with low in vitro cross-reactivity (≤ 1:5) to anti-AAV8, to deliver mouse activated Factor VII (mFVIIa)...
May 6, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28462595/intranasal-aav-mediated-gene-delivery-and-expression-of-human-iduronidase-in-the-cns-a-non-invasive-and-effective-approach-for-prevention-of-neurologic-disease-in-mucopolysaccharidosis-type-i
#11
Lalitha R Belur, Alexa Temme, Kelly M Podetz-Pedersen, Maureen Riedl, Lucy Vulchanova, Nicholas Robinson, Leah R Hanson, Karen F Kozarsky, Paul J Orchard, William H Frey Ii, Walter C Low, R Scott McIvor
Mucopolysaccharidosis type I (MPS I) is a progressive, multisystemic, inherited metabolic disease caused by deficiency of -L-iduronidase (IDUA). Current treatments for this disease are ineffective in treating CNS disease due to the inability of lysosomal enzymes to traverse the blood-brain barrier. We have taken a non-invasive and effective approach to the treatment of CNS disease by intranasal administration of an IDUA-encoding adeno-associated virus serotype 9 (AAV9) vector. Adult IDUA-deficient mice were immunotolerized at birth with human iduronidase (Aldurazyme), to prevent anti-IDUA immune response, and at 3 months of age were instilled intranasally with AAV9-IDUA vector...
May 2, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28447859/transgene-expression-in-dogs-after-liver-directed-hydrodynamic-delivery-of-i-sleeping-beauty-i-transposons-using-balloon-catheters
#12
Kendra Anne Hyland, Elena L Aronovich, Erik R Olson, Jason B Bell, Myra Urness Rusten, Roland Gunther, David W Hunter, Perry B Hackett, R Scott McIvor
The <i>Sleeping Beauty</i> transposon system has been extensively tested for integration of reporter and therapeutic genes <i>in vitro</i> and <i>in vivo</i> in mice. Dogs were used as a large animal model for human therapy and minimally invasive infusion of DNA solutions. DNA solutions were delivered into the entire liver or the left side of the liver using balloon catheters for temporary occlusion of venous outflow. A peak intravascular pressure between 80 and 140 mmHg supported sufficient DNA delivery in dog liver for detection of secretable reporter proteins...
April 27, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28446032/effect-of-intravesical-liposome-based-nerve-growth-factor-antisense-therapy-on-bladder-overactivity-and-nociception-in-a-rat-model-of-cystitis-induced-by-hydrogen-peroxide
#13
Tsuyoshi Majima, Pradeep Tyagi, Koji Dogishi, Mahendra Kashyap, Yasuhito Funahashi, Momokazu Gotoh, Michael B Chancellor, Naoki Yoshimura
The aim of this study was to evaluate whether liposome-based local suppression of nerve growth factor (NGF) in the bladder has effects on bladder hypersensitivity in a rat cystitis model induced by intravesical instillation of hydrogen peroxide (HP). HP (1.5%) was intravesically administered to adult female Sprague-Dawley rats. Liposomes complexed with NGF antisense oligonucleotide (OND) labeled with TYE563 fluorescent tag were intravesically instilled on day 2. Red fluorescence from the TYE 563 tag was observed with fluorescent microscopy on day 3...
April 18, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28345428/induction-of-t-cell-infiltration-and-programmed-death-ligand-2-expression-by-adeno-associated-virus-in-rhesus-macaque-skeletal-muscle-and-modulation-by-prednisone
#14
Megan L Cramer, Guohong Shao, Louise R Rodino-Klapac, Louis G Chicoine, Paul T Martin
Use of adeno-associated virus (AAV) to transduce genes into skeletal muscles can be associated with T-cell responses to viral capsid and/or to transgenic protein. Intramuscular mononuclear cell infiltrates primarily consisting of CD8+ T cells and also containing FOXP3+ regulatory T cells were present in rhesus macaque skeletal muscle treated with rAAVrh74.MCK.GALGT2 by vascular delivery. Administration of oral prednisone prior to AAV gene delivery and throughout the study reduced such infiltrates by 60% at 24 weeks post AAV delivery compared with AAV-treated animals not receiving prednisone, regardless of the presence of pre-existing AAV serum antibodies at the time of treatment...
March 23, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28335619/overexpression-of-the-x-linked-inhibitor-of-apoptosis-protects-against-retinal-degeneration-in-a-feline-model-of-retinal-detachment
#15
Sarah J Wassmer, Brian C Leonard, Stuart G Coupland, Adam N Baker, John Hamilton, William W Hauswirth, Catherine Tsilfidis
Retinal detachment is an acute disorder in humans that is caused by trauma or disease, and it can often lead to permanent visual deficits that result from the death of photoreceptors in the retina. The final pathway for photoreceptor cell death is apoptosis and necroptosis. The X-linked inhibitor of apoptosis (XIAP) has been shown to block both of these cell death pathways. This study tested the effects of XIAP on photoreceptor survival in a feline model of retinal detachment. The study was performed in 12 cats, divided into two experimental groups...
March 23, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28264583/crispr-mediated-knockout-of-cybb-in-nsg-mice-establishes-a-model-of-chronic-granulomatous-disease-for-human-stem-cell-gene-therapy-transplants
#16
Colin L Sweeney, Uimook Choi, Chengyu Liu, Sherry Koontz, Seung-Kwon Ha, Harry L Malech
Chronic granulomatous disease (CGD) is characterized by defects in the production of microbicidal reactive oxygen species (ROS) by phagocytes. Testing of gene and cell therapies for the treatment of CGD in human hematopoietic cells requires preclinical transplant models. The use of the lymphocyte-deficient NOD.Cg-Prkdc(scid) Il2rg(tm1Wjl/)SzJ (NSG) mouse strain for human hematopoietic cell xenografts to test CGD therapies is complicated by the presence of functional mouse granulocytes capable of producing ROS for subsequent bacterial and fungal killing...
March 6, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28498777/authors-response-to-jesse-d-riordan-hum-gene-ther-2017-28-375-376-doi-10-1089-hum-2017-045
#17
Arun Srivastava, Barrie J Carter
No abstract text is available yet for this article.
May 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28498776/aav-capsid-engineering-zooming-in-on-the-target
#18
Thierry VandenDriessche
No abstract text is available yet for this article.
May 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28363247/gene-delivery-of-calreticulin-anti-angiogenic-domain-attenuates-the-development-of-choroidal-neovascularization-in-rats
#19
Youn-Shen Bee, Leilei Tu, Shwu-Jiuan Sheu, Hsiu-Chen Lin, Jia-Hua Tang, Jiang-Hui Wang, Selwyn M Prea, Gregory J Dusting, Deng-Chyang Wu, Jingxiang Zhong, Bang V Bui, Ming-Hong Tai, Guei-Sheung Liu
Choroidal neovascularization (CNV) is a common pathological feature in neovascular age-related macular degeneration, which is the leading cause of vision loss among elderly populations in developed countries. This study evaluated the effect of a novel endogenous inhibitor of angiogenesis, calreticulin anti-angiogenic domain (CAD), subconjunctivally delivered by an adenoviral vector (Ad-CAD) in a rat model of laser-induced CNV. CAD was expressed in Ad-CAD-infected cells and inhibited the angiogenic activity in human umbilical vein endothelial cells in vitro...
May 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28335621/re-srivastava-a-carter-bj-aav-infection-protection-from-cancer-hum-gene-ther-2017-28-323-327-doi-10-1089-hum-2016-147
#20
Jesse D Riordan
No abstract text is available yet for this article.
May 2017: Human Gene Therapy
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