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"Gene therapy"

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https://www.readbyqxmd.com/read/28748410/cytotoxic-effect-of-co-expression-of-human-hepatitis-a-virus-3c-protease-and-bifunctional-suicide-protein-fcu1-genes-in-a-bicistronic-vector
#1
Alexey Komissarov, Ilya Demidyuk, Dina Safina, Marina Roschina, Andrey Shubin, Nataliya Lunina, Maria Karaseva, Sergey Kostrov
Recent reports on various cancer models demonstrate a great potential of cytosine deaminase/5-fluorocytosine suicide system in cancer therapy. However, this approach has limited success and its application to patients has not reached the desirable clinical significance. Accordingly, the improvement of this suicide system is an actively developing trend in gene therapy. The purpose of this study was to explore the cytotoxic effect observed after co-expression of hepatitis A virus 3C protease (3C) and yeast cytosine deaminase/uracil phosphoribosyltransferase fusion protein (FCU1) in a bicistronic vector...
July 26, 2017: Molecular Biology Reports
https://www.readbyqxmd.com/read/28748310/long-term-outcome-of-adenosine-deaminase-deficient-patients-a-single-center-experience
#2
Ori Scott, Vy Hong-Diep Kim, Brenda Reid, Anne Pham-Huy, Adelle R Atkinson, Alessandro Aiuti, Eyal Grunebaum
PURPOSE: Inherited defects in the adenosine deaminase (ADA) enzyme can cause severe combined immune deficiency (SCID) and systemic abnormalities. Management options for ADA-deficient patients include enzyme replacement therapy (ERT), hematopoietic stem cell transplantation (HSCT), and gene therapy (GT). Here, we describe the long-term benefits of these treatments. METHODS: Survival, infections, systemic sequelae, and laboratory assessments were recorded for all ADA-deficient SCID patients, managed at a single center since 1985, who survived 5 or more years following treatment...
July 26, 2017: Journal of Clinical Immunology
https://www.readbyqxmd.com/read/28748212/genetically-engineered-multilineage-differentiating-stress-enduring-cells-as-cellular-vehicles-against-malignant-gliomas
#3
Tomohiro Yamasaki, Shohei Wakao, Hiroshi Kawaji, Shinichiro Koizumi, Tetsuro Sameshima, Mari Dezawa, Hiroki Namba
Malignant glioma, the most common malignant brain tumor in adults, is difficult to treat due to its aggressive invasive nature. Enzyme/prodrug suicide gene therapy based on the herpes simplex virus thymidine kinase (HSVtk)/ganciclovir (GCV) system is an efficient strategy for treating malignant gliomas. In the present study, we evaluated treatment with multilineage-differentiating stress-enduring (Muse) cells, which are endogenous non-tumorigenic pluripotent-like stem cells that are easily collectable from the bone marrow as SSEA-3(+) cells, as carriers of the HSVtk gene...
September 15, 2017: Molecular Therapy Oncolytics
https://www.readbyqxmd.com/read/28747638/puma-gene-delivery-to-synoviocytes-reduces-inflammation-and-degeneration-of-arthritic-joints
#4
Saw-See Hong, Hubert Marotte, Guillaume Courbon, Gary S Firestein, Pierre Boulanger, Pierre Miossec
In rheumatoid arthritis (RA), the proliferation of fibroblast-like synoviocytes (FLS) is the cause of chronic inflammation in joints and of joint damage. Delivery of the pro-apoptotic gene PUMA to FLS via human adenovirus type 5 (HAdV5) vectors has been tested as a therapeutic approach, but efficiency is hampered by low transduction, as FLS do not express HAdV5 receptors on the cell surface. Here we show that efficient transduction of PUMA in FLS can be achieved by conjugating HAdV5 to a baculovirus, which binds to the cell surface via the envelope glycoprotein Gp64...
July 27, 2017: Nature Communications
https://www.readbyqxmd.com/read/28747142/development-of-the-first-who-lentiviral-vector-standard-towards-the-production-control-and-standardisation-of-lentivirus-based-gene-therapy-products
#5
Yuan Zhao, Hannah Stepto, Christian K Schneider
Gene therapy is a rapidly evolving field; so far, there have been more than 2400 gene therapy products in clinical trials and four products on the market (http://www.abedia.com/wiley/vectors.php). A prerequisite for producing gene therapy products is ensuring their quality and safety. This requires appropriately controlled and standardized production and testing procedures that result in consistent safety and efficacy. Assuring the quality and safety of lentivirus based gene therapy products in particular presents a great challenge because they are cell-based multi-gene products which include viral and therapeutic proteins as well as modified cells...
July 26, 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/28744947/bmp-2-gene-activated-muscle-tissue-fragments-for-osteochondral-defect-regeneration-in-the-rabbit-knee
#6
Volker M Betz, Alexander Keller, Peter Foehr, Christian Thirion, Michael Salomon, Stefan Rammelt, Hans Zwipp, Rainer Burgkart, Volkmar Jansson, Peter E Müller, Oliver B Betz
BACKGROUND: Previously published data indicate that BMP-2 gene activated muscle tissue grafts can repair large bone defects in rats. This innovative abbreviated ex vivo gene therapy is appealing since it does not require elaborative and time-consuming extraction and expansion of cells. Hence, in the here presented experimental study we evaluated the potential of this expedited tissue engineering approach to regenerate osteochondral defects in rabbits. METHODS: Autologous muscle tissue grafts from female White New Zealand rabbits were directly transduced with an adenoviral BMP-2 vector or remained unmodified...
July 25, 2017: Journal of Gene Medicine
https://www.readbyqxmd.com/read/28743034/therapeutic-advances-in-musculoskeletal-aav-targeting-approaches
#7
REVIEW
Karen Bulaklak, Xiao Xiao
The use of recombinant adeno-associated viruses (rAAVs) is highly prevalent in musculoskeletal gene therapies due to their versatility, high transduction efficiency, natural tropism and vector genome persistence for years. As the largest organ in the body, treatment of skeletal muscle for widespread and sufficient therapeutic gene expression is highly challenging. In addition to disease-specific hurdles, vector genome loss, off-target gene transfer and immune responses to treatment can diminish the overall benefit of rAAV therapies...
July 22, 2017: Current Opinion in Pharmacology
https://www.readbyqxmd.com/read/28742067/long-term-microdystrophin-gene-therapy-is-effective-in-a-canine-model-of-duchenne-muscular-dystrophy
#8
Caroline Le Guiner, Laurent Servais, Marie Montus, Thibaut Larcher, Bodvaël Fraysse, Sophie Moullec, Marine Allais, Virginie François, Maeva Dutilleul, Alberto Malerba, Taeyoung Koo, Jean-Laurent Thibaut, Béatrice Matot, Marie Devaux, Johanne Le Duff, Jack-Yves Deschamps, Inès Barthelemy, Stéphane Blot, Isabelle Testault, Karim Wahbi, Stéphane Ederhy, Samia Martin, Philippe Veron, Christophe Georger, Takis Athanasopoulos, Carole Masurier, Federico Mingozzi, Pierre Carlier, Bernard Gjata, Jean-Yves Hogrel, Oumeya Adjali, Fulvio Mavilio, Thomas Voit, Philippe Moullier, George Dickson
Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Gene therapy using highly functional microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors is an attractive strategy to treat DMD. Here we show that locoregional and systemic delivery of a rAAV2/8 vector expressing a canine microdystrophin (cMD1) is effective in restoring dystrophin expression and stabilizing clinical symptoms in studies performed on a total of 12 treated golden retriever muscular dystrophy (GRMD) dogs...
July 25, 2017: Nature Communications
https://www.readbyqxmd.com/read/28741493/derisking-drug-induced-carcinogenicity-for-novel-therapeutics
#9
REVIEW
Jonathan G Moggs, Timothy MacLachlan, Hans-Joerg Martus, Philip Bentley
Assessing the carcinogenic potential of innovative drugs spanning diverse therapeutic modalities and target biology represents a major challenge during drug development. Novel modalities, such as cell and gene therapies that involve intrinsic genetic modification of the host genome, require distinct approaches for identification of cancer hazard. We emphasize the need for customized weight-of-evidence cancer risk assessments based on mode of action that balance multiple options for preclinical identification of cancer hazard with appropriate labeling of clinical products and risk management plans...
August 2016: Trends in Cancer
https://www.readbyqxmd.com/read/28741454/nanovesicular-formulations-for-cancer-gene-therapy
#10
Lorena Tavano, Elisabetta Mazzotta, Rita Muzzalupo
In the last decades, gene therapy has become a novel therapeutic strategy for cancer treatment, including immunologic and molecular approaches. Among molecular avenue, the design of efficient and effective gene delivery systems, like cationic liposomes and niosomes, has been widely investigated and proposed as the most promising research area. The advantages of cationic vesicles rely on their natural ability to form complexes with anionic genetic molecules and deliver them into the cells via the endosomal pathway...
May 18, 2017: Current Pharmaceutical Design
https://www.readbyqxmd.com/read/28741230/new-therapeutic-strategies-for-lewy-body-dementias
#11
REVIEW
Latha Velayudhan, Dominic Ffytche, Clive Ballard, Dag Aarsland
This article reviews current treatment strategies and recent advances for the Lewy body dementias (LBDs). Current available symptom treatment strategies are based on monoaminergic, cholinergic and glutaminergic neurotransmitter systems. Relatively robust evidence exists for cholinesterase inhibitors for cognitive impairment in LBD and in Parkinson's disease for antidepressants, clozapine and recently pimavanserin for psychosis. interpidine (RVT 101) and nelotanserin are currently under investigation. Non-pharmacological interventions, such as cognitive stimulation, physical exercises and neuromodulation strategies, may be useful in Parkinson's disease but have not yet been tested in dementias...
September 2017: Current Neurology and Neuroscience Reports
https://www.readbyqxmd.com/read/28740654/a-novel-therapeutic-approach-for-esophageal-squamous-cell-carcinoma-suppressor-of-cytokine-signaling-1-gene-therapy
#12
EDITORIAL
Chang-Han Chen, Shau-Hsuan Li
No abstract text is available yet for this article.
June 2017: Journal of Thoracic Disease
https://www.readbyqxmd.com/read/28739283/the-pharmacology-of-gene-therapy
#13
EDITORIAL
Seppo Ylä-Herttuala
No abstract text is available yet for this article.
July 21, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28737744/delivering-advanced-therapies-the-big-pharma-approach
#14
REVIEW
J Tarnowski, D Krishna, L Jespers, A Ketkar, R Haddock, J Imrie, S Kili
After two decades of focused development and some recent clinical successes, cell and gene therapy (CGT) is emerging as a promising approach to personalized medicines. Genetically engineered cells as a medical modality are poised to stand alongside or in combination with small molecule and biopharmaceutical approaches to bring new therapies to patients globally. Big pharma can play a vital role in industrializing CGT by focusing on diseases with high un-met medical need and compelling genetic evidence. Pharma should invest in manufacturing and supply chain solutions that deliver reproducible, high quality therapies at a commercially viable cost...
July 24, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28737743/development-of-gene-therapies-lessons-from-nusinersen
#15
REVIEW
L Xu, I Irony, W W Bryan, B Dunn
The nusinersen development and approval process provide important lessons regarding the pathway to marketing approval for gene therapies. These lessons emphasize rigorous clinical trial design, flexibility in trial design and analysis, a collaborative effort with regular communications between the drug developer and the FDA, and use of FDA's expedited programs. These lessons are critical to the development of gene therapies for the treatment of serious or life-threatening rare diseases.Gene Therapy accepted article preview online, 24 July 2017...
July 24, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28737023/monodispersed-bioactive-glass-nanoclusters-with-ultralarge-pores-and-intrinsic-exceptionally-high-mirna-loading-for-efficiently-enhancing-bone-regeneration
#16
Yumeng Xue, Yi Guo, Meng Yu, Min Wang, Peter X Ma, Bo Lei
Bioactive glass nanoparticles (BGNs) have attracted much attention in drug delivery and bone tissue regeneration, due to the advantages including biodegradation, high bone-bonding bioactivity, and facile large-scale fabrication. However, the wide biomedical applications of BGNs such as efficient gene delivery are limited due to their poor pore structure and easy aggregation. Herein, for the first time, this study reports novel monodispersed bioactive glass nanoclusters (BGNCs) with ultralarge mesopores (10-30 nm) and excellent miRNA delivery for accelerating critical-sized bone regeneration...
July 24, 2017: Advanced Healthcare Materials
https://www.readbyqxmd.com/read/28733131/preclinical-studies-for-a-phase-1-clinical-trial-of-autologous-hematopoietic-stem-cell-gene-therapy-for-sickle-cell-disease
#17
Fabrizia Urbinati, Jennifer Wherley, Sabine Geiger, Beatriz Campo Fernandez, Michael L Kaufman, Aaron Cooper, Zulema Romero, Filippo Marchioni, Lilith Reeves, Elizabeth Read, Barbara Nowicki, Elke Grassman, Shivkumar Viswanathan, Xiaoyan Wang, Roger P Hollis, Donald B Kohn
BACKGROUND AIMS: Gene therapy by autologous hematopoietic stem cell transplantation (HSCT) represents a new approach to treat sickle cell disease (SCD). Optimization of the manufacture, characterization and testing of the transduced hematopoietic stem cell final cell product (FCP), as well as an in depth in vivo toxicology study, are critical for advancing this approach to clinical trials. METHODS: Data are shown to evaluate and establish the feasibility of isolating, transducing with the Lenti/β(AS3)-FB vector and cryopreserving CD34(+) cells from human bone marrow (BM) at clinical scale...
July 18, 2017: Cytotherapy
https://www.readbyqxmd.com/read/28731526/drug-discovery-and-development-for-rare-genetic-disorders
#18
REVIEW
Wei Sun, Wei Zheng, Anton Simeonov
Approximately 7,000 rare diseases affect millions of individuals in the United States. Although rare diseases taken together have an enormous impact, there is a significant gap between basic research and clinical interventions. Opportunities now exist to accelerate drug development for the treatment of rare diseases. Disease foundations and research centers worldwide focus on better understanding rare disorders. Here, the state-of-the-art drug discovery strategies for small molecules and biological approaches for orphan diseases are reviewed...
July 21, 2017: American Journal of Medical Genetics. Part A
https://www.readbyqxmd.com/read/28731177/co%C3%A2-expression-of-axin-and-apc-gene-fragments-inhibits-colorectal-cancer-cell-growth-via-regulation-of-the-wnt-signaling-pathway
#19
Meili Xu, Xianling Liu, Yan Xu, Shicong Zhu, Yawen Gao
Adenomatous polyposis coli (APC) and Axin interactions serve an important role in colorectal cancer (CRC) pathogenesis. The aim of the present study was to assess the combined effects of Axin and APC co‑expression in CRC cells, and to determine the underlying mechanisms involved. SW480 cells were divided into the following groups: Untransfected (SW480 group), transfected with pEGFP‑N3plus pCS2‑MT (SW480/vector‑vector), transfected with pEGFP‑N3‑APC5 (SW480/APC5), and transfected with pEGFP‑N3‑APC5 pluspCS2‑MT‑Axin (SW480/APC5‑Axin)...
July 21, 2017: Molecular Medicine Reports
https://www.readbyqxmd.com/read/28731040/er-stress-and-the-unfolded-protein-response-in-neurodegeneration
#20
REVIEW
Claudio Hetz, Smita Saxena
The clinical manifestation of neurodegenerative diseases is initiated by the selective alteration in the functionality of distinct neuronal populations. The pathology of many neurodegenerative diseases includes accumulation of misfolded proteins in the brain. In physiological conditions, the proteostasis network maintains normal protein folding, trafficking and degradation; alterations in this network - particularly disturbances to the function of endoplasmic reticulum (ER) - are thought to contribute to abnormal protein aggregation...
July 21, 2017: Nature Reviews. Neurology
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