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https://www.readbyqxmd.com/read/29783206/-experimental-evaluation-of-the-therapeutic-potential-of-liposome-mediated-apoe3-gene-transfection-for-cerebral-atherosclerosis
#1
Vadym V Biloshytskyi, Alisa V Pachevska, Alina V Biloshytska, Valerii M Istoshyn, Maryna V Biloshytska, Olha B Shevnia
OBJECTIVE: Introduction: In recent years, a stroke has been the cause of high lethality, long-term and sustained disability, the problem of which is still far from being resolved. The root cause of stroke is atherosclerosis. The aim: Study of neurotrophic and neuroprotective functions of apoE in rat brain tissue in experimental atherosclerosis. PATIENTS AND METHODS: Materials and methods: The study included 60 sexually mature rats. The model of experimental atherosclerosis was created by Anichkov method...
2018: Wiadomości Lekarskie: Organ Polskiego Towarzystwa Lekarskiego
https://www.readbyqxmd.com/read/29783100/cd133-cells-derived-from-skeletal-muscles-of-duchenne-muscular-dystrophy-patients-have-a-compromised-myogenic-and-muscle-regenerative-capability
#2
Jinhong Meng, Francesco Muntoni, Jennifer Morgan
Cell-mediated gene therapy is a possible means to treat muscular dystrophies like Duchenne muscular dystrophy. Autologous patient stem cells can be genetically-corrected and transplanted back into the patient, without causing immunorejection problems. Regenerated muscle fibres derived from these cells will express the missing dystrophin protein, thus improving muscle function. CD133+ cells derived from normal human skeletal muscle contribute to regenerated muscle fibres and form muscle stem cells after their intra-muscular transplantation into an immunodeficient mouse model...
May 12, 2018: Stem Cell Research
https://www.readbyqxmd.com/read/29782834/high-throughput-dissection-of-aav-host-interactions-the-fast-and-the-curious
#3
REVIEW
Anne-Kathrin Herrmann, Dirk Grimm
Over fifty years after its initial description, Adeno-associated virus (AAV) remains a most exciting but also most elusive study object in basic or applied virology. On the one hand, its simple structure not only facilitates investigations into virus biology, but combined with the availability of numerous natural AAV variants with distinct infection efficiency and specificity also makes AAV a preferred substrate for engineering of gene delivery vectors. On the other hand, it is striking to witness a recent flurry of reports that highlight and partially close persistent gaps in our understanding of AAV virus and vector biology...
May 18, 2018: Journal of Molecular Biology
https://www.readbyqxmd.com/read/29782345/precision-surgery-for-obesity
#4
Zhiyong Dong, Sheikh Mohammed Shariful Islam, Enan Simms-Walker, Cunchuan Wang
Precision medicine is targeted towards improving the effectiveness of treatment, reducing the side effects of drugs and reducing medical costs. The application of precision surgery for obesity is a new concept that involves 2 stages: the first stage is to attain a precise obesity surgery, and the second stage is to achieve individualized obese gene therapy. In this article, we discuss the value of precision surgery for obesity, its stages and its future application to improve obesity surgery. Due to recent advancements in medical technologies, genetics, surgical and clinical research; precision surgery for obesity will lead the future of obesity surgery...
May 16, 2018: American Journal of Therapeutics
https://www.readbyqxmd.com/read/29781995/an-ecdysone-receptor-based-singular-gene-switch-for-deliberate-expression-of-transgene-with-robustness-reversibility-and-negligible-leakiness
#5
Seoghyun Lee, Minho Won, Ran Hee Hwang, Gang Min Hur, Hyunju Ro
Precise control of transgene expression is desirable in biological and clinical studies. However, because the binary feature of currently employed gene switches requires the transfer of two therapeutic expression units concurrently into a single cell, the practical application of the system for gene therapy is limited. To simplify the transgene expression system, we generated a gene switch designated as pEUI(+) encompassing a complete set of transgene expression modules in a single vector. Comprising of the GAL4 DNA-binding domain and modified EcR (GvEcR), a minimal VP16 activation domain fused with a GAL4 DNA-binding domain, as well as a modified Drosophila ecdysone receptor (EcR), the newly developed singular gene switch is highly responsive to the administration of a chemical inducer in a time- and dosage-dependent manner...
May 7, 2018: Journal of Visualized Experiments: JoVE
https://www.readbyqxmd.com/read/29781327/the-progress-of-aav-mediated-gene-therapy-in-neuromuscular-disorders
#6
Sara Aguti, Alberto Malerba, Haiyan Zhou
The well-defined genetic causes and monogenetic nature of many neuromuscular disorders, including Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), present gene therapy as a prominent therapeutic approach. The novel variants of adeno-associated virus (AAV) can achieve satisfactory transduction efficiency of exogenous genes through the central nervous system and body-wide in skeletal muscle. Areas covered: In this review, we summarize the strategies of AAV gene therapy that are currently under preclinical and clinical evaluation for the treatment of degenerative neuromuscular disorders, with a focus on diseases such as DMD and SMA...
May 20, 2018: Expert Opinion on Biological Therapy
https://www.readbyqxmd.com/read/29781145/core-outcome-set-for-gene-therapy-in-haemophilia-results-of-the-corehem-multistakeholder-project
#7
A Iorio, M W Skinner, E Clearfield, D Messner, G F Pierce, M Witkop, S Tunis
BACKGROUND: Gene therapy trial results show potential to cure haemophilia A and haemophilia B. Securing broad access to a cure for a lifelong chronic disease is anticipated to face barriers at the individual and healthcare system levels, which can be partly mitigated by harmonized planning of clinical research studies. The aim of the coreHEM project was to determine the set of outcome measures required to evaluate efficacy, safety, comparative effectiveness and value of gene therapy for haemophilia...
May 20, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29781034/specific-inhibitor-of-notch%C3%A2-3-enhances-the-sensitivity-of-nsclc-cells-to-gemcitabine
#8
Bi-Dan Hu, Jia Guo, Yuan-Zi Ye, Ting Du, Chun-Song Cheng, Qian Jiang, Ruo-Nan Liu, Yan-Bei Zhang
Notch‑3 is a receptor of the Notch signaling pathway and plays an important role in regulating self‑renewal, differentiation and apoptosis in cancer cells. Overexpression of Notch‑3 has been proved to be associated with resistance to gemcitabine (GEM) and poor patient prognosis for various malignant tumors. In the present study, two non‑small cell lung cancer (NSCLC) cell lines, H1299 and A549, were induced with GEM for two months and then were treated with various concentrations of a Notch signaling blocker, N‑[N‑(3,5‑difluorophenacetyl)‑L‑alanyl]‑S‑phenylglycine t‑butyl ester (DAPT), with the goal of reducing expression of Notch intracellular domain 3 (NICD3)...
May 16, 2018: Oncology Reports
https://www.readbyqxmd.com/read/29779339/-research-advances-on-gene-therapy-for-hemophilia
#9
X Y Dai, L Zhang
No abstract text is available yet for this article.
April 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29779176/intranasal-delivery-of-pgdnf-dna-nanoparticles-provides-neuroprotection-in-the-rat-6-hydroxydopamine-model-of-parkinson-s-disease
#10
Amirah E-E Aly, Brendan T Harmon, Linas Padegimas, Ozge Sesenoglu-Laird, Mark J Cooper, Barbara L Waszczak
Glial cell line-derived neurotrophic factor (GDNF) gene therapy could offer a disease-modifying treatment for Parkinson's disease (PD). Here, we report that plasmid DNA nanoparticles (NPs) encoding human GDNF administered intranasally to rats induce transgene expression in the brain and protect dopamine neurons in a model of PD. To first test whether intranasal administration could transfect cells in the brain, rats were sacrificed 1 week after intranasal pGDNF NPs or the naked plasmid. GDNF ELISA revealed significant increases in GDNF expression throughout the brain for both treatments...
May 19, 2018: Molecular Neurobiology
https://www.readbyqxmd.com/read/29777875/switching-cell-penetrating-and-cxcr4-binding-activities-of-nanoscale-organized-arginine-rich-peptides
#11
Marianna Teixeira de Pinho Favaro, Naroa Serna, Laura Sánchez-García, Rafael Cubarsi, Mónica Roldán, Alejandro Sánchez-Chardi, Ugutz Unzueta, Ramón Mangues, Neus Ferrer-Miralles, Adriano Rodrigues Azzoni, Esther Vázquez, Antonio Villaverde
Arginine-rich protein motifs have been described as potent cell-penetrating peptides (CPPs) but also as rather specific ligands of the cell surface chemokine receptor CXCR4, involved in the infection by the human immunodeficiency virus (HIV). Polyarginines are commonly used to functionalize nanoscale vehicles for gene therapy and drug delivery, aimed to enhance cell penetrability of the therapeutic cargo. However, under which conditions these peptides do act as either unspecific or specific ligands is unknown...
May 16, 2018: Nanomedicine: Nanotechnology, Biology, and Medicine
https://www.readbyqxmd.com/read/29776328/recent-progress-and-considerations-for-aav-gene-therapies-targeting-the-central-nervous-system
#12
REVIEW
Erik Allen Lykken, Charles Shyng, Reginald James Edwards, Alejandra Rozenberg, Steven James Gray
BACKGROUND: Neurodevelopmental disorders, as a class of diseases, have been particularly difficult to treat even when the underlying cause(s), such as genetic alterations, are understood. What treatments do exist are generally not curative and instead seek to improve quality of life for affected individuals. The advent of gene therapy via gene replacement offers the potential for transformative therapies to slow or even stop disease progression for current patients and perhaps minimize or prevent the appearance of symptoms in future patients...
May 18, 2018: Journal of Neurodevelopmental Disorders
https://www.readbyqxmd.com/read/29775572/gene-therapy-with-antisense-oligonucleotides-silencing-c-myc-reduces-neointima-formation-and-vessel-wall-thickness-in-a-mouse-model-of-vein-graft-disease
#13
Christina Maria Steger, Nikolaos Bonaros, Ralf Joachim Rieker, Johannes Bonatti, Thomas Schachner
Gene therapy for avoiding intimal hyperplasia of vein grafts after coronary artery bypass grafting is still discussed controversially. A promising application of gene therapy in vein grafts is the use of antisense oligonucleotides to block the expression of genes encoding cell cycle regulatory proteins in vascular smooth muscle cells. C-myc, either directly or by regulating the expression of other proteins, controls cell proliferation, apoptosis and cell survival, tissue remodeling, angiogenesis, cell metabolism, production of inflammatory and anti-inflammatory cytokines, and also participates in cell transformation...
May 15, 2018: Experimental and Molecular Pathology
https://www.readbyqxmd.com/read/29774076/the-human-somatostatin-receptor-type-2-as-an-imaging-and-suicide-reporter-gene-for-pluripotent-stem-cell-derived-therapy-of-myocardial-infarction
#14
Katrien Neyrinck, Natacha Breuls, Bryan Holvoet, Wouter Oosterlinck, Esther Wolfs, Hubert Vanbilloen, Olivier Gheysens, Robin Duelen, Willy Gsell, Ivo Lambrichts, Uwe Himmelreich, Catherine M Verfaillie, Maurilio Sampaolesi, Christophe M Deroose
Rationale: Pluripotent stem cells (PSCs) are being investigated as a cell source for regenerative medicine since they provide an infinitive pool of cells that are able to differentiate towards every cell type of the body. One possible therapeutic application involves the use of these cells to treat myocardial infarction (MI), a condition where billions of cardiomyocytes (CMs) are lost. Although several protocols have been developed to differentiate PSCs towards CMs, none of these provide a completely pure population, thereby still posing a risk for neoplastic teratoma formation...
2018: Theranostics
https://www.readbyqxmd.com/read/29774063/dual-target-gene-therapy-to-eml4-alk-nsclc-by-a-gold-nanoshell-based-system
#15
Siwen Li, Yuxi Liu, Yalan Rui, Liping Tang, Samuel Achilefu, Yueqing Gu
Although EML4-ALK transforming fusion gene is represented in only 8% of non-small cell lung cancer (NSCLC) cases, its expression is partly responsive for the failure of current NSCLC treatments. Preventing secondary mutation of the ALK protein through direct gene manipulation could overcome NSCLC drug resistance. Method: In this study, we developed a gold nanoshell (HAuNs) drug carrier for delivery and selective photo-thermal release of genes that target ALK and microRNA-301 in NSCLC. Additionally, the densely-coated nanoshell adsorbed high amounts of the positively-charged anticancer drug doxorubicin (DOX), generating an exciting multidimensional treatment strategy that includes gene-, thermal- and chemo- therapy...
2018: Theranostics
https://www.readbyqxmd.com/read/29772345/autologous-liquid-platelet-rich-fibrin-a-novel-drug-delivery-system
#16
REVIEW
Richard J Miron, Yufeng Zhang
There is currently widespread interest within the biomaterial field to locally deliver biomolecules for bone and cartilage regeneration. Substantial work to date has focused on the potential role of these biomolecules during the healing process, and the carrier system utilized is a key factor in their effectiveness. Platelet rich fibrin (PRF) is a naturally derived fibrin scaffold that is easily obtained from peripheral blood following centrifugation. Slower centrifugation speeds have led to the commercialization of a liquid formulation (liquid-PRF) resulting in an upper plasma layer composed of liquid fibrinogen/thrombin prior to clot formation that remains in its liquid phase for approximately 15 minutes until injected into bodily tissues...
May 14, 2018: Acta Biomaterialia
https://www.readbyqxmd.com/read/29771464/degradation-of-amyloid-%C3%AE-peptide-by-neprilysin-expressed-from-borna-disease-virus-vector
#17
Madoka Sakai, Sakiho Ueda, Takuji Daito, Megumi Asada-Utsugi, Yumiko Komatsu, Ayae Kinoshita, Takakuni Maki, Akira Kuzuya, Ryosuke Takahashi, Akiko Makino, Keizo Tomonaga
Accumulation of amyloid β (Aβ40 and Aβ42) in the brain is a characteristic of Alzheimer's disease (AD). Because neprilysin (NEP) is a major Aβ-degrading enzyme, NEP delivery in the brain is a promising gene therapy for AD. Borna disease virus (BoDV) vector enables long-term transduction of foreign genes in the central nerve system. Here, we evaluated the proteolytic ability of NEP transduced by the BoDV vector and found that the amounts of Aβ40 and Aβ42 significantly decreased, which suggests that NEP expressed from the BoDV vector is functional to degrade Aβ...
May 17, 2018: Microbiology and Immunology
https://www.readbyqxmd.com/read/29770199/emerging-therapies-for-hemophilia-controversies-and-unanswered-questions
#18
REVIEW
Valder R Arruda, Bhavya S Doshi, Benjamin J Samelson-Jones
Several new therapies for hemophilia have emerged in recent years. These strategies range from extended half-life factor replacement products and non-factor options with improved pharmacokinetic profiles to gene therapy aiming for phenotypic cure. While these products have the potential to change hemophilia care dramatically, several challenges and questions remain regarding broader applicability, long-term safety, and which option to pursue for each patient. Here, we review these emerging therapies with a focus on controversies and unanswered questions in each category...
2018: F1000Research
https://www.readbyqxmd.com/read/29769001/editorial-current-gene-therapy-on-non-viral-gene-delivery-and-therapy
#19
EDITORIAL
Lihua Peng
No abstract text is available yet for this article.
2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29769000/preface-translational-gene-therapy-coming-of-age
#20
Lung-Ji Chang, Alfred S Lewin
No abstract text is available yet for this article.
2018: Current Gene Therapy
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