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Human Gene Therapy

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https://www.readbyqxmd.com/read/28622065/leptin-gene-transfer-improves-symptoms-of-type-2-diabetic-mice-by-regulating-leptin-signaling-pathway-and-insulin-resistance-of-peripheral-tissues
#1
Lan Xiang, Jing Li, Qian Wang, Ruiqi Tang, Jianhua Qi
The leptin gene was transferred into the liver of streptozocin- and high fat diet-induced type 2 diabetic (T2D) mice by hydrodynamic-based gene delivery. The food intake, water consumption, glucose concentration, and triglyceride, total cholesterol levels of T2D mice were significantly decreased. Meanwhile, plasma leptin was remarkably increased after gene transfer for 2, 3, 5, and 7 days, while plasma adiponectin was also significantly increased at day 2. To understand the mechanism of action of leptin on T2D mice, gene expressions related to glycometabolism and energy metabolism in the liver, epididymal adipose tissue, hypothalamus, and muscle were measured...
June 16, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28599597/simultaneous-knockout-of-i-cxcr4-i-and-i-ccr5-i-genes-in-cd4-t-cells-via-crispr-cas9-confers-resistance-to-both-x4-and-r5-tropic-hiv-1-infection
#2
Songlin Yu, Yongchao Yao, Hongkui Xiao, Jiaojiao Li, Quan Liu, Yijun Yang, Dickson Adah, Junnan Lu, Siting Zhao, Li Qin, Xiaoping Chen
Previous research has proven that disruption of either <i>CCR5</i> or <i>CXCR4</i> gene confers resistance to R5-tropic or X4-tropic HIV-1 infection, respectively. However, the urgent need to ablate both of the coreceptors in individual post-thymic CD4+ T cells for dual protection remains. Here, we have ablated the <i>CCR5</i> and <i>CXCR4</i> genes in human CD4+ cell lines and primary CD4+ T cells, simultaneously, using CRISPR/Cas9, a well-developed, highly efficient genetic engineering tool...
June 9, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28578603/aav-gene-therapy-for-alcoholism-inhibition-of-mitochondrial-aldehyde-dehydrogenase-enzyme-expression-in-hepatoma-cells
#3
Anamaria C Sanchez, Chengwen Li, Barbara A Andrews, Juan A Asenjo, R Jude Samulski
Most of the ethanol is broken down in the liver, through two-step by Alcohol dehydrogenase (ADH) and Aldehyde dehydrogenase (ALDH2) enzymes, which metabolized down ethanol into acetaldehyde and then acetate. Some individuals of the Asian population who carry a mutation in the Aldehyde dehydrogenase gene (ALDH2*2) cannot metabolize acetaldehyde as efficiently, producing strong effects including facial flushing, dizziness, hypotension, and palpitations. This results in an aversion to alcohol intake and protection against alcoholism...
June 3, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28557574/the-hydrophobic-effect-from-conjugated-chemicals-or-drugs-on-in-vivo-biodistribution-of-rna-nanoparticles
#4
Daniel L Jasinski, Hongran Yin, Zhefeng Li, Peixuan Guo
liver or other organ accumulation of drugs is one of the major problems that leads to toxicity and side effects in therapy using chemicals or other macromolecules. It has been shown that specially designed RNA nanoparticles can specifically target cancer cells, silence oncogenic genes, and stop cancer growth with little or no accumulation in the liver or other vital organs. It is well known that physical properties of nanoparticles such as size, shape, and surface chemistry affect biodistribution and pharmacokinetic profiles <i></i>in vivo<i></i>...
May 30, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28557533/induced-cell-turnover-a-novel-therapeutic-modality-for-in-situ-tissue-regeneration
#5
Francesco Albert Bosco Cortese, Sebastian Aguiar, Giovanni Santostasi
Induced Cell Turnover (ICT) is a theoretical intervention in which the targeted ablation of damaged, diseased and/or nonfunctional cells is coupled with replacement by partially differentiated induced pluripotent stem cells in a gradual and multi-phasic manner. Tissue-specific ablation can be achieved using pro-apoptotic small molecule cocktails, peptide mimetics, and/or tissue-tropic AAV-delivered suicide genes driven by cell-type specific promoters. Replenishment with new cells can be mediated by systemic administration of cells engineered for homing, robustness, and even enhanced function and disease resistance...
May 30, 2017: Human Gene Therapy
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
#6
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
#7
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
#8
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
#9
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
#10
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
#11
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
#12
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/28478695/prevention-of-neurocognitive-deficiency-in-mucopolysaccharidosis-type-ii-mice-by-cns-directed-aav9-mediated-iduronate-sulfatase-gene-transfer
#13
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/28478735/gene-therapy-of-adult-neuronal-ceroid-lipofuscinoses-with-crispr-cas9-in-zebrafish
#14
Xiaomin Yao, Xiaowei Liu, Yaguang Zhang, Yuhao Li, Chenjian Zhao, Shaohua Yao, Yuquan 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 the DNAJC5 gene that encodes cysteine-string protein alpha (CSPα) have been reported to be associated with familial autosomal-dominant ANCL (AD-ANCL). This study constructed an ANCL transgenic zebrafish model expressing the human mutant DNAJC5 (mDNAJC5) gene under the control of a zebrafish neuron-specific promoter...
May 5, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28478688/gene-delivery-of-activated-factor-vii-using-alternative-adeno-associated-virus-serotype-improves-hemostasis-in-hemophiliac-mice-with-fviii-inhibitors-and-adeno-associated-virus-neutralizing-antibodies
#15
Junjiang Sun, Baolai Hua, Xiaojing Chen, Richard J 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, AAV9, a serotype with low in vitro cross-reactivity (≤1:5) to anti-AAV8, was used to deliver mouse-activated factor VII (mFVIIa)...
May 5, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28462595/intranasal-adeno-associated-virus-mediated-gene-delivery-and-expression-of-human-iduronidase-in-the-central-nervous-system-a-noninvasive-and-effective-approach-for-prevention-of-neurologic-disease-in-mucopolysaccharidosis-type-i
#16
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, multi-systemic, inherited metabolic disease caused by deficiency of α-L-iduronidase (IDUA). Current treatments for this disease are ineffective in treating central nervous system (CNS) disease due to the inability of lysosomal enzymes to traverse the blood-brain barrier. A noninvasive and effective approach was taken in the treatment of CNS disease by intranasal administration of an IDUA-encoding adeno-associated virus serotype 9 (AAV9) vector. Adult IDUA-deficient mice aged 3 months were instilled intranasally with AAV9-IDUA vector...
April 20, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28447859/transgene-expression-in-dogs-after-liver-directed-hydrodynamic-delivery-of-sleeping-beauty-transposons-using-balloon-catheters
#17
Kendra A 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 Sleeping Beauty transposon system has been extensively tested for integration of reporter and therapeutic genes in vitro and in vivo 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 19, 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
#18
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/28604173/one-more-controversy-adeno-associated-virus-in-stem-cells
#19
Terence R Flotte
No abstract text is available yet for this article.
June 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
#20
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...
June 2017: Human Gene Therapy
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