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https://www.readbyqxmd.com/read/28320116/in-vitro-non-viral-murine-pro-neurotrophin-3-gene-transfer-into-rat-bone-marrow-stromal-cells
#1
Shahram Darabi, Taki Tiraihi, AliReza Delshad, Majid Sadeghizadeh, Wisam Khalil, Taher Taheri
Neurotrophin 3 (NT-3) is an important factor for promoting prenatal neural development, as well as regeneration, axogenesis and plasticity in postnatal life. Therapy with NT-3 was reported to improve the condition of patients suffering from degenerative diseases and traumatic injuries, however, the disadvantage of NT-3 protein delivery is its short half-life, thus our alternative approach is the use of NT-3 gene therapy. In this study, the bone marrow stromal cells (BMSCs) were isolated from adult rats, cultured for 4 passages and transfected with either pEGFP-N1 or a constructed vector containing murine proNT-3 (pSecTag2/HygroB-murine proNT-3) using Lipofectamine 2000 followed by Hygromycin B (200mg/kg)...
April 15, 2017: Journal of the Neurological Sciences
https://www.readbyqxmd.com/read/28319449/non-clinical-study-examining-aav8-tbg-hldlr-vector-associated-toxicity-in-chow-fed-wild-type-and-ldlr-rhesus-macaques
#2
Jenny A Greig, Maria P Limberis, Peter Bell, Shu-Jen Chen, Roberto Calcedo, Daniel J Rader, James M Wilson
Vectors based on adeno-associated virus serotype 8 (AAV8) have been evaluated in several clinical trials of gene therapy for hemophilia B with encouraging results. In preparation for a Phase 1 clinical trial of AAV8 gene therapy for the treatment of homozygous familial hypercholesterolemia (HoFH), the safety of the clinical candidate vector, AAV8.TBG.hLDLR, was evaluated in wild-type rhesus macaques and macaques heterozygous for a nonsense mutation in the low-density lipoprotein receptor (LDLR) gene (LDLR(+/-))...
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28319447/gene-therapy-briefs
#3
Alex Philippidis
No abstract text is available yet for this article.
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28319446/good-laboratory-practice-preclinical-safety-studies-for-gsk2696273-mlv-vector-based-ex-vivo-gene-therapy-for-adenosine-deaminase-deficiency-severe-combined-immunodeficiency-in-nsg-mice
#4
Nicola Carriglio, Jan Klapwijk, Raisa Jofra Hernandez, Michela Vezzoli, Franck Chanut, Rhiannon Lowe, Draghici Elena, Melanie Nord, Paola Albertini, Patrizia Cristofori, Jane Richards, Hazel Staton, Jonathan Appleby, Alessandro Aiuti, Aisha V Sauer
GSK2696273 (autologous CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase [ADA] enzyme) is a gamma-retroviral ex vivo gene therapy of bone marrow-derived CD34+ cells for the treatment of adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID). ADA-SCID is a severe monogenic disease characterized by immunologic and nonimmunologic symptoms. Bone-marrow transplant from a matched related donor is the treatment of choice, but it is available for only a small proportion of patients...
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28319445/nonclinical-pharmacology-toxicology-study-of-aav8-tbg-mldlr-and-aav8-tbg-hldlr-in-a-mouse-model-of-homozygous-familial-hypercholesterolemia
#5
Jenny A Greig, Maria P Limberis, Peter Bell, Shu-Jen Chen, Roberto Calcedo, Daniel J Rader, James M Wilson
The homozygous form of familial hypercholesterolemia (HoFH) is an excellent model for developing in vivo gene therapy in humans. The success of orthotropic liver transplantation in correcting the metabolic abnormalities in HoFH suggests that the correction of low-density lipoprotein receptor (LDLR) expression in hepatocytes via gene therapy should be sufficient for therapeutic efficacy. Vectors based on adeno-associated virus serotype 8 (AAV8) have been previously developed for liver-targeted gene therapy of a number of genetic diseases, including HoFH...
March 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28319444/retro-orbital-venous-sinus-delivery-of-raav9-mediates-high-level-transduction-of-brain-and-retina-compared-with-temporal-vein-delivery-in-neonatal-mouse-pups
#6
Alisha M Gruntman, Lin Su, Terence R Flotte
In order to pursue a clinical gene therapy for a human neurologic disease, it is often necessary to perform proof-of-concept trials in mouse models of that disease. In order to demonstrate a potential clinical efficacy, one must be able to select an appropriate vector and route of delivery for the appropriate age group in the disease model. Since many diseases require correction early in life, investigators often need to deliver recombinant adeno-associated viral (rAAV) vectors to neonatal mice. Herein, general central nervous system expression patterns of nuclear GFP following delivery of rAAV by three different routes are reported...
March 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28319443/the-negative-effects-of-immigration-restrictions-on-the-gene-therapy-community
#7
Terence R Flotte
No abstract text is available yet for this article.
March 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28318721/-physiology-of-the-visual-retinal-signal-from-phototransduction-to-the-visual-cycle
#8
REVIEW
C Salesse
The retinal photoreceptors (rods and cones) are responsible for light absorption and transduction of the signal, which is transmitted to the other retinal nerve cells and then to the brain. The chromophore of visual pigments of rods and cones is a particular isomer of a vitamin A derivative. Light absorption by this chromophore leads to its isomerization and to a phototransduction cascade, which results in photoreceptor hyperpolarization and cessation of glutamate secretion at their synaptic terminals. Phototransduction of cones and rods differs in their signal amplification and inactivation, which is consistent with their respective functions...
March 17, 2017: Journal Français D'ophtalmologie
https://www.readbyqxmd.com/read/28318165/-effects-of-rhoa-silencing-on-proliferation-of-tongue-squamous-cancer-cells
#9
Yan Guoxin, Fan Bing, Zou Ronghai, Zhang Jian, Sun Xiaofeng, Tong Lei, Wang Qimin, Han Jinhong, Lu Xufei, Wang Ying, Zhou Yuan, He Zongxuan, Liao Yixiang, Li Ning, Cao Lei, Chen Zhenggang
OBJECTIVE: This study investigated the effect of RhoA silencing through RNA interference on proliferation and growth of tongue cancer cells, as well as explored the possible mechanisms of this effect. METHODS: SSC-4 tongue cancer cells were cultured in vitro and then transfected with small interfering RNA to knock down RhoA expression. The tested cells were divided into three groups: experimental group (experimental group 1: transfected with RhoA-siRNA-1; experi-mental group 2: transfected with RhoA-siRNA-2), negative control group (transfected by random sequence NC-siRNA), and blank control group (transfected with Lipofectamine)...
December 1, 2016: Hua Xi Kou Qiang Yi Xue za Zhi, Huaxi Kouqiang Yixue Zazhi, West China Journal of Stomatology
https://www.readbyqxmd.com/read/28318036/autophagy-determines-efficiency-of-liver-directed-gene-therapy-with-adeno-associated-viral-vectors
#10
Marianna Hösel, Anke Huber, Susanne Bohlen, Julie Lucifora, Giuseppe Ronzitti, Francesco Puzzo, Florence Boisgerault, Ulrich T Hacker, Wilhelmus J Kwanten, Nora Klöting, Matthias Blüher, Alexander Gluschko, Michael Schramm, Olaf Utermöhlen, Wilhelm Bloch, Federico Mingozzi, Oleg Krut, Hildegard Büning
Use of Adeno-associated viral (AAV) vectors for liver-directed gene therapy has shown considerable success, particularly in patients with severe hemophilia B. However, the high vector doses required to reach therapeutic levels of transgene expression caused liver inflammation in some patients that selectively destroyed transduced hepatocytes. We hypothesized that such detrimental immune responses can be avoided by enhancing the efficacy of AAV vectors in hepatocytes. Because autophagy is a key liver response to environmental stresses, we characterized the impact of hepatic autophagy on AAV infection...
March 20, 2017: Hepatology: Official Journal of the American Association for the Study of Liver Diseases
https://www.readbyqxmd.com/read/28317871/insights-into-molecular-therapy-of-glioma-current-challenges-and-next-generation-blueprint
#11
REVIEW
Y Rajesh, Ipsita Pal, Payel Banik, Sandipan Chakraborty, Sachin A Borkar, Goutam Dey, Ahona Mukherjee, Mahitosh Mandal
Glioma accounts for the majority of human brain tumors. With prevailing treatment regimens, the patients have poor survival rates. In spite of current development in mainstream glioma therapy, a cure for glioma appears to be out of reach. The infiltrative nature of glioma and acquired resistance substancially restrict the therapeutic options. Better elucidation of the complicated pathobiology of glioma and proteogenomic characterization might eventually open novel avenues for the design of more sophisticated and effective combination regimens...
March 20, 2017: Acta Pharmacologica Sinica
https://www.readbyqxmd.com/read/28317361/-analysis-of-bnip3-expression-and-clinical-research-in-salivary-adenoid-cystic-carcinoma
#12
Chen Zhanwei, Sun Dubin, Huang Shengyun, Wu Haiwei, Zhang Dongsheng
OBJECTIVE: This study investigated the expression of BNIP3 in salivary adenoid cystic carcinoma (SACC) and its correlations to the clinicopathological features and prognosis of patients with SACC. The role of BNIP3 in the progress of hypoxia-induced autophagy was elucidated. METHODS: The expression levels of BNIP3, hypoxia inducible factor (HIF)-1α, and LC3 in 65 SACC cases were detected by immunohistochemical staining method, and the correlation between the expression of BNIP3 and the clinicopathological features in SACC was analyzed...
August 1, 2016: Hua Xi Kou Qiang Yi Xue za Zhi, Huaxi Kouqiang Yixue Zazhi, West China Journal of Stomatology
https://www.readbyqxmd.com/read/28316197/-inhibitory-effect-of-insulin-like-growth-factor-binding-protein-related-protein-1-on-retinal-angiogenesis-via-erk-signaling-pathway
#13
P Zhang, H Wang, H Cao, X Xu, T Sun
Objective: To explore the inhibitory effect of insulin-like growth factor binding protein-related protein 1 (IGFBP-rP1), a novel anti-angiogenic factor, on retinal angiogenesis and its underlying molecular mechanisms. Methods: Experimental study. C57BL/6J mice were classified into three groups: control group (n=24), oxygen-induced retinopathy (OIR) non-intervention group (n=24) and OIR intervention group (n=72). The OIR mouse model was established using improved Smith's methods (n= 96). Twelve-day-old mice in the OIR intervention group were randomly assigned into three groups receiving intravitreal injection of recombinant mouse IGFBP-rP1 (50 μg/L, 100 μg/L and 200 μg/L, respectively)...
March 11, 2017: [Zhonghua Yan Ke za Zhi] Chinese Journal of Ophthalmology
https://www.readbyqxmd.com/read/28315384/the-absorption-enhancer-sodium-deoxycholate-promotes-high-gene-transfer-in-skeletal-muscles
#14
Christian Leborgne, Debborah Alimi-Guez, Nelly El Shafey, Laetitia van Wittenberghe, Pascal Bigey, Daniel Scherman, Antoine Kichler
Gene delivery to skeletal muscle is a promising strategy for the treatment of muscle disorders and for the systemic secretion of therapeutic proteins. In addition, muscle is an attractive target tissue because it is easily accessible. However, very few synthetic vectors proved capable of surpassing naked DNA mediated muscle gene transfer. In fact, only neutral copolymers, in particular poloxamers, demonstrated capacities to increase transgene expression in skeletal muscles. Here, we studied in vitro and in vivo behaviour of different bile salts...
March 14, 2017: International Journal of Pharmaceutics
https://www.readbyqxmd.com/read/28315379/magnetic-nanoparticles-for-efficient-cell-transduction-with-semliki-forest-virus
#15
Baiba Kurena, Aleksandra Vežāne, Dace Skrastiņa, Olga Trofimova, Anna Zajakina
Semliki Forest virus (SFV) is a potential cancer gene therapy vector capable of providing high and transient expression of heterologous proteins in mammalian cells. However, SFV has shown suboptimal transduction levels in several cancer cell types as well as wide biodistribution of SFV has been observed after in vivo applications. Magnetic nanoparticles (MNPs) have been shown to increase cell transduction with several viral vectors in vitro under an external magnetic field and enhance magnetically guided viral vector delivery...
March 14, 2017: Journal of Virological Methods
https://www.readbyqxmd.com/read/28314988/polymeric-micelles-as-mighty-nanocarriers-for-cancer-gene-therapy-a-review
#16
REVIEW
Mahsa Yousefpour Marzbali, Ahmad Yari Khosroushahi
PURPOSE: Gene therapy has shown extensive potential to treat human diseases occurring from the defection of genes like various types of cancers. The cationic polymers, as non-viral gene carriers, offer the ability to engineer carrier systems having customized features that can be adapted to suit any system. Upon polymeric micelle systems' core-shell structure, micelles can create the capacity to load genes/gene-drugs into the different micelle compartments, respectively. METHODS: The search will be managed in Pubmed, Medline, Cochrane library, Embase and Proquest for articles related to polymeric micelle-based gene delivery in order to cancer gene therapy using the accommodative search terms...
March 17, 2017: Cancer Chemotherapy and Pharmacology
https://www.readbyqxmd.com/read/28314976/enzyme-replacement-therapy-and-beyond-in-memoriam-roscoe-o-brady-m-d-1923-2016
#17
REVIEW
Markus Ries
Lysosomal storage disorders are strong candidates for the development of specific innovative therapies. The discovery of enzyme deficiencies is an important milestone in understanding the underlying cause of disease. Being able to replace the first missing enzyme in a lysosomal storage required three decades of dedicated research. Successful drug development for lysosomal storage disorders was fostered by the U.S. Orphan Drug Act. Various optimization strategies have the potential to overcome the current limitations of enzyme replacement therapies...
March 17, 2017: Journal of Inherited Metabolic Disease
https://www.readbyqxmd.com/read/28314668/extracellular-superoxide-dismutase-increased-the-therapeutic-potential-of-human-mesenchymal-stromal-cells-in-radiation-pulmonary-fibrosis
#18
Li Wei, Jing Zhang, Zai-Liang Yang, Hua You
BACKGROUND AIMS: Pulmonary fibrosis induced by irradiation is a significant problem of radiotherapy in cancer patients. Extracellular superoxide dismutase (SOD3) is found to be predominantly and highly expressed in the extracellular matrix of lung and plays a pivotal role against oxidative damage. Early administration of mesenchymal stromal cells (MSCs) has been demonstrated to reduce fibrosis of damaged lung. However, injection of MSCs at a later stage would be involved in fibrosis development...
March 14, 2017: Cytotherapy
https://www.readbyqxmd.com/read/28314351/current-strategies-in-cancer-gene-therapy
#19
(no author information available yet)
No abstract text is available yet for this article.
March 2017: Anticancer Research
https://www.readbyqxmd.com/read/28314212/gene-delivery-of-tipe2-inhibits-breast-cancer-development-and-metastasis-via-cd8-t-and-nk-cell-mediated-antitumor-responses
#20
Zhenhua Zhang, Li Liu, Shousong Cao, Yizhun Zhu, Qibing Mei
Breast cancer is the second leading cause of cancer-related deaths in the female patients which was mainly caused by metastasis. Development of target gene therapy for breast cancer to suppress tumor progress and metastasis will improve the therapeutic options and be of great benefit to the patients. Tumor necrosis factor-alpha-induced protein 8-like 2 is a novel molecule for maintaining immune homeostasis and is involved in cancer development. In the present study, we overexpressed TIPE2 in breast cancer cells to investigate the role of TIPE2 in the development of breast cancer...
March 14, 2017: Molecular Immunology
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