keyword
MENU ▼
Read by QxMD icon Read
search

Gene-therapy

keyword
https://www.readbyqxmd.com/read/28213069/a-pilot-study-of-cell-mediated-gene-therapy-for-spinal-cord-injury-in-mini-pigs
#1
Islamov Rustem Robertovich, Sokolov Mikhail Evgenyevich, Bashirov Farid Vagizovich, Fadeev Filip Olegovich, Shmarov Maxim Michaylovich, Naroditskiy Boris Savelyevich, Povysheva Tatyana Vyacheslavovna, Shaymardanova Gulnara Ferdinantovna, Yakupov Radik Albertovich, Chelyshev Yuri Aleksandrovich, Lavrov Igor Aleksandrovich
Currently, in clinical practice there is no efficient way to overcome the sequences of neurodegeneration after spinal cord traumatic injury. Using a new experimental model of spinal cord contusion injury on miniature pigs, we proposed to deliver therapeutic genes encoding vascular endothelial growth factor (VEGF), glial cell line-derived neurotrophic factor (GDNF) and neural cell adhesion molecule (NCAM) to the damaged area, using umbilical cord blood mononuclear cells (UCBC). In this study, genetically engineered UCBC (2×10(6) cells in 200 ml of saline) were injected intrathecally to mini-pigs 10days after SCI...
February 14, 2017: Neuroscience Letters
https://www.readbyqxmd.com/read/28212271/carbon-nanotubes-as-an-effective-opportunity-for-cancer-diagnosis-and-treatment
#2
REVIEW
Alessandro Sanginario, Beatrice Miccoli, Danilo Demarchi
Despite the current progresses of modern medicine, the resistance of malignant tumors to present medical treatments points to the necessity of developing new therapeutic approaches. In recent years, numerous studies have focused their attention on the promising use of nanomaterials, like iron oxide nanowires, zinc oxide or mesoporous silica nanoparticles, for cancer and metastasis treatment with the advantage of operating directly at the bio-molecular scale. Among them, carbon nanotubes emerged as valid candidates not only for drug delivery, but also as a valuable tool in cancer imaging and physical ablation...
February 15, 2017: Biosensors
https://www.readbyqxmd.com/read/28209901/vitamin-b3-modulates-mitochondrial-vulnerability-and-prevents-glaucoma-in-aged-mice
#3
Pete A Williams, Jeffrey M Harder, Nicole E Foxworth, Kelly E Cochran, Vivek M Philip, Vittorio Porciatti, Oliver Smithies, Simon W M John
Glaucomas are neurodegenerative diseases that cause vision loss, especially in the elderly. The mechanisms initiating glaucoma and driving neuronal vulnerability during normal aging are unknown. Studying glaucoma-prone mice, we show that mitochondrial abnormalities are an early driver of neuronal dysfunction, occurring before detectable degeneration. Retinal levels of nicotinamide adenine dinucleotide (NAD(+), a key molecule in energy and redox metabolism) decrease with age and render aging neurons vulnerable to disease-related insults...
February 17, 2017: Science
https://www.readbyqxmd.com/read/28208742/aav-vector-mediated-gene-delivery-to-substantia-nigra-dopamine-neurons-implications-for-gene-therapy-and-disease-models
#4
REVIEW
Katrina Albert, Merja H Voutilainen, Andrii Domanskyi, Mikko Airavaara
Gene delivery using adeno-associated virus (AAV) vectors is a widely used method to transduce neurons in the brain, especially due to its safety, efficacy, and long-lasting expression. In addition, by varying AAV serotype, promotor, and titer, it is possible to affect the cell specificity of expression or the expression levels of the protein of interest. Dopamine neurons in the substantia nigra projecting to the striatum, comprising the nigrostriatal pathway, are involved in movement control and degenerate in Parkinson's disease...
February 8, 2017: Genes
https://www.readbyqxmd.com/read/28208635/advances-in-non-viral-dna-vectors-for-gene-therapy
#5
Cinnamon L Hardee, Lirio Milenka Arévalo-Soliz, Benjamin D Hornstein, Lynn Zechiedrich
Uses of viral vectors have thus far eclipsed uses of non-viral vectors for gene therapy delivery in the clinic. Viral vectors, however, have certain issues involving genome integration, the inability to be delivered repeatedly, and possible host rejection. Fortunately, development of non-viral DNA vectors has progressed steadily, especially in plasmid vector length reduction, now allowing these tools to fill in specifically where viral or other non-viral vectors may not be the best options. In this review, we examine the improvements made to non-viral DNA gene therapy vectors, highlight opportunities for their further development, address therapeutic needs for which their use is the logical choice, and discuss their future expansion into the clinic...
February 10, 2017: Genes
https://www.readbyqxmd.com/read/28206989/il-4-mediated-by-hsv-vector-suppresses-morphine-withdrawal-response-through-decreasing-tnf%C3%AE-nr2b-and-pc-ebp%C3%AE-in-the-periaqueductal-gray-in-rats
#6
H Yi, T Iida, S Liu, D Ikegami, Q Liu, A Iida, D A Lubarsky, S Hao
Chronic opiates induce the development of physical dependence. Opioid physical dependence characterized by withdrawal symptoms, may have very long-lasting effects on the motivation for reward, including the incubation of cue-induced drug-seeking behavior. Elucidation of the mechanisms involved in physical dependence is crucial to developing more effective treatment strategies for opioid dependence. Chronic morphine induces production of proinflammatory cytokines in regional-specific sites of the brain. Interleukin-4 (IL-4) is a prototypical anti-inflammatory cytokine that globally suppresses proinflammatory cytokines...
February 16, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28206750/synthetic-riboswitches-from-plug-and-pray-towards-plug-and-play
#7
Maja Etzel, Mario Mörl
In synthetic biology, metabolic engineering as well as gene therapy there is a strong demand for orthogonal or externally controlled regulation of gene expression. Here, RNA-based regulatory devices represent a promising emerging alternative to proteins, allowing a fast and direct control of gene expression, as no synthesis of regulatory proteins is required. Besides programmable ribozyme elements controlling mRNA stability, regulatory RNA structures in untranslated regions are highly interesting for engineering approaches...
February 16, 2017: Biochemistry
https://www.readbyqxmd.com/read/28204850/-biological-therapies-in-otology-german-version
#8
A Roemer, H Staecker, S Sasse, T Lenarz, A Warnecke
Millions of people worldwide suffer from hearing loss. Current treatment for patients with severe to profound hearing loss consists of cochlear implants. Providing the cochlear nerve is intact, patients generally benefit enormously from this intervention, frequently achieving significant improvements in speech comprehension. There are, however, some cases where current technology does not provide patients with adequate benefit. New therapeutic concepts based on cell transplantation and gene therapy are developing rapidly, at least in the research sector...
February 15, 2017: HNO
https://www.readbyqxmd.com/read/28203188/endoscope-assisted-and-controlled-argus-ii-epiretinal-prosthesis-implantation-in-late-stage-retinitis-pigmentosa-a-report-of-2-cases
#9
Emin Özmert, Sibel Demirel
Several different approaches for restoring sight in subjects who are blind due to outer retinal degeneration are currently under investigation, including stem cell therapy, gene therapy, and visual prostheses. Although many different types of visual prostheses have shown promise, to date, the Argus II Epiretinal Prosthesis System, developed in a clinical setting over the course of 10 years, is the world's first and only retinal prosthesis that has been approved by the United States Food and Drug Administration (FDA) and has been given the CE-Mark for sale within the European Economic Area (EEA)...
September 2016: Case Reports in Ophthalmology
https://www.readbyqxmd.com/read/28202824/-disease-modifying-therapy-for-parkinson-s-disease
#10
Hideki Shimura, Nobutaka Hattori
Currently, treatment of Parkinson's disease aims at alleviating its symptoms. However development of disease-modifying drugs has been a remarkable advancement in recent years. Furthermore, clinical trials of immunotherapy against α-synuclein, a protein involved in the pathogenesis of and lesion expansion in Parkinson's disease, have been initiated. Here, the disease-modifying treatment for patients with Parkinson's disease including the current α-synuclein immunotherapy, gene therapy, protein injection therapy, and cell transplantation therapy, has been reviewed...
February 2017: Brain and Nerve, Shinkei Kenkyū No Shinpo
https://www.readbyqxmd.com/read/28202760/restriction-of-hcmv-replication-by-isg15-a-host-effector-regulated-by-cgas-sting-dsdna-sensing
#11
Christopher Bianco, Ian Mohr
Accumulation of the interferon-stimulated gene (ISG) 15 protein product, which is reversibly conjugated to numerous polypeptide targets, impacts the proteome and physiology of uninfected and infected cells. While many viruses, including human cytomegalovirus (HCMV) blunt host antiviral defenses by limiting ISG expression, the overall abundance of ISG15 monomer and protein conjugates rises in HCMV-infected cells. However, the molecular signals underlying ISG15 accumulation and whether the ISG15 polypeptide itself influences HCMV infection biology remain unknown...
February 15, 2017: Journal of Virology
https://www.readbyqxmd.com/read/28202235/stx12-lncrna-mir-148a-smad5-participate-in-the-regulation-of-pancreatic-stellate-cell-activation-through-a-mechanism-involving-competing-endogenous-rna
#12
Hao Wang, Yanfeng Jiang, Ming Lu, Bei Sun, Xin Qiao, Dongbo Xue, Weihui Zhang
BACKGROUND: With the deepening of research, the roles of LncRNAs play in the fibrotic process have attracted great attention. At the early stage of pancreatic fibrogenesis, to effectively regulate pancreatic stellate cells (PSCs) activation is crucial for the treatment of chronic pancreatic fibrosis. METHODS: Microarray data on chronic pancreatitis were retrieved from the Gene Expression Omnibus (GEO) repository and analyzed using bioinformatic methods. A diagram of the lncRNA-miRNA-mRNA ceRNA network was constructed...
February 1, 2017: Pancreatology: Official Journal of the International Association of Pancreatology (IAP) ... [et Al.]
https://www.readbyqxmd.com/read/28199112/mitochondrial-gene-therapy-advances-in-mitochondrial-gene-cloning-plasmid-production-and-nanosystems-targeted-to-mitochondria
#13
Eduarda Coutinho, Cátia Batista, Fani Sousa, João Queiroz, Diana Costa
Mitochondrial gene therapy seems to be a valuable and promising strategy to treat mitochondrial disorders. The use of a therapeutic vector based on mitochondrial DNA, along with its affinity to the site of mitochondria can be considered a powerful tool in the reestablishment of normal mitochondrial function. In line with this and for the first time, we successfully cloned the mitochondrial gene ND1 that was stably maintained in multi-copy pCAG-GFP plasmid, used to transform E. coli. This mitochondrial gene based plasmid was encapsulated into nanoparticles...
February 15, 2017: Molecular Pharmaceutics
https://www.readbyqxmd.com/read/28195400/hypersonic-poration-a-new-versatile-cell-poration-method-to-enhance-cellular-uptake-using-a-piezoelectric-nano-electromechanical-device
#14
Zhixin Zhang, Yanyan Wang, Hongxiang Zhang, Zifan Tang, Wenpeng Liu, Yao Lu, Zefang Wang, Haitao Yang, Wei Pang, Hao Zhang, Daihua Zhang, Xuexin Duan
Efficient delivery of genes and therapeutic agents to the interior of the cell is critical for modern biotechnology. Herein, a new type of chemical-free cell poration method-hypersonic poration-is developed to improve the cellular uptake, especially the nucleus uptake. The hypersound (≈GHz) is generated by a designed piezoelectric nano-electromechanical resonator, which directly induces normal/shear stress and "molecular bombardment" effects on the bilayer membranes, and creates reversible temporal nanopores improving the membrane permeability...
February 13, 2017: Small
https://www.readbyqxmd.com/read/28194746/stimuli-responsive-polymeric-nanocarriers-for-efficient-gene-delivery
#15
REVIEW
Yingqin Li, Jinbiao Gao, Chao Zhang, Zhong Cao, Du Cheng, Jie Liu, Xintao Shuai
Gene therapy provides an alternative and effective method for treatment of genetic diseases and cancers that are refractory to conventional therapeutics. The success of gene therapy is largely dependent on the development of safe and effective gene delivery vectors for transporting genetic material from the blood stream to the cytoplasm or nucleus. Current gene vectors can be divided into viral and non-viral vectors. Although non-viral gene delivery carriers can offer some advantages, such as safety and facile fabrication, they do not possess the same high gene transfection efficiency as viral vectors due to a lack of functionality to overcome extra- and intracellular gene delivery obstacles...
April 2017: Topics in Current Chemistry (Journal)
https://www.readbyqxmd.com/read/28194615/how-we-manage-adenosine-deaminase-deficient-severe-combined-immune-deficiency-ada-scid
#16
Donald B Kohn, H Bobby Gaspar
Adenosine deaminase-deficient severe combined immune deficiency (ADA SCID) accounts for 10-15% of cases of human SCID. From what was once a uniformly fatal disease, the prognosis for infants with ADA SCID has improved greatly based on the development of multiple therapeutic options, coupled with more frequent early diagnosis due to implementation of newborn screening for SCID. We review the various treatment approaches for ADA SCID including allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen-matched sibling or family member or from a matched unrelated donor or a haplo-identical donor, autologous HSCT with gene correction of the hematopoietic stem cells (gene therapy-GT), and enzyme replacement therapy (ERT) with polyethylene glycol-conjugated adenosine deaminase...
February 14, 2017: Journal of Clinical Immunology
https://www.readbyqxmd.com/read/28193911/eif3i-activity-is-critical-for-endothelial-cells-in-tumor-induced-angiogenesis-through-regulating-vegfr-and-erk-translation
#17
Yaguang Zhang, Ping Wang, Qian Zhang, Xiaomin Yao, Linjie Zhao, Yibin Liu, Xiaowei Liu, Rui Tao, Chuan Yu, Yuhao Li, Xiangrong Song, Shaohua Yao
Translational control is a critical step in the regulation of gene expression. Accumulating evidence shows that translational control of a subgroup of mRNAs tends to be selective. However, our understanding of the function of selective translational control in endothelial cells is still incomplete. We found that a key translational regulator, eIF3i, is highly expressed in endothelial cells during embryonic and tumor angiogenesis. Knockdown of eIF3i restrained cell proliferation and migration in endothelial cells...
February 11, 2017: Oncotarget
https://www.readbyqxmd.com/read/28193196/rapid-eradication-of-colon-carcinoma-by-clostridium-perfringens-enterotoxin-suicidal-gene-therapy
#18
Jessica Pahle, Lutz Menzel, Nicole Niesler, Dennis Kobelt, Jutta Aumann, Maria Rivera, Wolfgang Walther
BACKGROUND: Bacterial toxins have evolved to an effective therapeutic option for cancer therapy. The Clostridium perfringens enterotoxin (CPE) is a pore-forming toxin with selective cytotoxicity. The transmembrane tight junction proteins claudin-3 and -4 are known high affinity CPE receptors. Their expression is highly upregulated in human cancers, including breast, ovarian and colon carcinoma. CPE binding to claudins triggers membrane pore complex formation, which leads to rapid cell death...
February 13, 2017: BMC Cancer
https://www.readbyqxmd.com/read/28193190/enhanced-expression-of-vastatin-inhibits-angiogenesis-and-prolongs-survival-in-murine-orthotopic-glioblastoma-model
#19
Yi Li, Jun Li, Yat Ming Woo, Zan Shen, Hong Yao, Yijun Cai, Marie Chia-Mi Lin, Wai Sang Poon
BACKGROUND: Antiangiogenic therapies are considered promising for the treatment of glioblastoma (GB). The non-collagenous C-terminal globular NC1 domain of type VIII collagen a1 chain, Vastatin, is an endogenous antiangiogenic polypeptide. Sustained enhanced expression of Vastatin was shown to inhibit tumour growth and metastasis in murine hepatocellular carcinoma models. In this study, we further explored the efficacy of Vastatin in the treatment of GB xenografts. METHOD: Treatment of Vastatin was carried out using a nanopolymer gene vector PEI600-CyD-Folate (H1)...
February 13, 2017: BMC Cancer
https://www.readbyqxmd.com/read/28193101/how-to-successfully-screen-random-aav-display-peptide-libraries-in-vivo
#20
Jakob Körbelin, Martin Trepel
Adeno-associated virus (AAV) has emerged as a very promising gene therapy vector. To enable tissue-directed gene expression, many artificially generated AAV variants have been established, often isolated from large pools of mutated capsids. Random peptide libraries displayed on AAV capsids have been used successfully to select vectors targeted to a given target cell or tissue in vitro and in vivo. However, the published methodology for screening of AAV libraries to isolate vectors with selective tissue tropism after intravenous administration in vivo has not been described in sufficient detail to address all critical steps...
February 14, 2017: Human Gene Therapy Methods
keyword
keyword
8055
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"