keyword
MENU ▼
Read by QxMD icon Read
search

Gene-therapy

keyword
https://www.readbyqxmd.com/read/27935605/evolution-of-the-circuitry-for-conscious-color-vision-in-primates
#1
J Neitz, M Neitz
There are many ganglion cell types and subtypes in our retina that carry color information. These have appeared at different times over the history of the evolution of the vertebrate visual system. They project to several different places in the brain and serve a variety of purposes allowing wavelength information to contribute to diverse visual functions. These include circadian photoentrainment, regulation of sleep and mood, guidance of orienting movements, detection and segmentation of objects. Predecessors to some of the circuits serving these purposes presumably arose before mammals evolved and different functions are represented by distinct ganglion cell types...
December 9, 2016: Eye
https://www.readbyqxmd.com/read/27934883/preclinical-evaluation-of-radiation-and-systemic-rgd-targeted-adeno-associated-virus-phage-tnf-gene-therapy-in-a-mouse-model-of-spontaneously-metastatic-melanoma
#2
T J Quinn, N Healy, A Sara, E Maggi, C S Claros, R Kabarriti, L Scandiuzzi, L Liu, J Gorecka, A Adem, I Basu, Z Yuan, C Guha
The incidence of melanoma in the United States continues to rise, with metastatic lesions notoriously recalcitrant to therapy. There are limited effective treatment options available and a great need for more effective therapies that can be rapidly integrated in the clinic. In this study, we demonstrate that the combination of RGD-targeted adeno-associated virus phage (RGD-AAVP-TNF) with hypofractionated radiation therapy results in synergistic inhibition of primary syngeneic B16 melanoma in a C57 mouse model...
December 9, 2016: Cancer Gene Therapy
https://www.readbyqxmd.com/read/27934882/stable-knockdown-of-creb-hif-1-and-hif-2-by-replication-competent-retroviruses-abrogates-the-responses-to-hypoxia-in-hepatocellular-carcinoma
#3
D Shneor, R Folberg, J Pe'er, A Honigman, S Frenkel
The fast proliferation of tumor cells develops faster than the vasculature, resulting, in most malignant tumors, in generation of hypoxic regions. Hypoxia renders solid tumors resistant to radiation and chemotherapeutics while providing opportunities for tumor-selective therapies targeting tumor hypoxia. Here we exploit two properties of tumors: propagation of tumor cells and ongoing generation of hypoxic regions to construct a system that preferentially leads to the death of tumor cells and thus hinders tumor growth...
December 9, 2016: Cancer Gene Therapy
https://www.readbyqxmd.com/read/27933309/impact-of-age-and-vector-construct-on-striatal-and-nigral-transgene-expression
#4
Nicole K Polinski, Fredric P Manfredsson, Matthew J Benskey, D Luke Fischer, Christopher J Kemp, Kathy Steece-Collier, Ivette M Sandoval, Katrina L Paumier, Caryl E Sortwell
Therapeutic protein delivery using viral vectors has shown promise in preclinical models of Parkinson's disease (PD) but clinical trial success remains elusive. This may partially be due to a failure to include advanced age as a covariate despite aging being the primary risk factor for PD. We investigated transgene expression following intracerebral injections of recombinant adeno-associated virus pseudotypes 2/2 (rAAV2/2), 2/5 (rAAV2/5), 2/9 (rAAV2/9), and lentivirus (LV) expressing green fluorescent protein (GFP) in aged versus young adult rats...
2016: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/27933308/tailored-transgene-expression-to-specific-cell-types-in-the-central-nervous-system-after-peripheral-injection-with-aav9
#5
Jonathan Dashkoff, Eli P Lerner, Nhi Truong, Jacob A Klickstein, Zhanyun Fan, Dakai Mu, Casey A Maguire, Bradley T Hyman, Eloise Hudry
The capacity of certain adeno-associated virus (AAV) vectors to cross the blood-brain barrier after intravenous delivery offers a unique opportunity for noninvasive brain delivery. However, without a well-tailored system, the use of a peripheral route injection may lead to undesirable transgene expression in nontarget cells or organs. To refine this approach, the present study characterizes the transduction profiles of new self-complementary AAV9 (scAAV9) expressing the green fluorescent protein (GFP) either under an astrocyte (glial fibrillary acidic (GFA) protein) or neuronal (Synapsin (Syn)) promoter, after intravenous injection of adult mice (2 × 10(13) vg/kg)...
2016: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/27933306/significant-changes-in-endogenous-retinal-gene-expression-assessed-1-year-after-a-single-intraocular-injection-of-aav-cntf-or-aav-bdnf
#6
Chrisna J LeVaillant, Anil Sharma, Jill Muhling, Lachlan Pg Wheeler, Greg S Cozens, Mats Hellström, Jennifer Rodger, Alan R Harvey
Use of viral vectors to deliver therapeutic genes to the central nervous system holds promise for the treatment of neurodegenerative diseases and neurotrauma. Adeno-associated viral (AAV) vectors encoding brain-derived neurotrophic factor (BDNF) or ciliary derived neurotrophic factor (CNTF) promote the viability and regeneration of injured adult rat retinal ganglion cells. However, these growth-inducing transgenes are driven by a constitutively active promoter, thus we examined whether long-term AAV-mediated secretion of BDNF or CNTF affected endogenous retinal gene expression...
2016: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/27933303/purification-of-baculovirus-vectors-using-heparin-affinity-chromatography
#7
Md Nasimuzzaman, Danielle Lynn, Johannes Cm van der Loo, Punam Malik
Baculoviruses are commonly used for recombinant protein and vaccine production. Baculoviruses are nonpathogenic to vertebrates, have a large packaging capacity, display broad host and cell type tropism, infect both dividing and nondividing cells, and do not elicit strong immune or allergic responses in vivo. Hence, their use as gene delivery vehicles has become increasingly popular in recent years. Moreover, baculovirus vectors carrying mammalian regulatory elements can efficiently transduce and express transgenes in mammalian cells...
2016: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/27933220/a-novel-method-for-quantitative-serial-autofluorescence-analysis-in-retinitis-pigmentosa-using-image-characteristics
#8
Jasleen K Jolly, Siegfried K Wagner, Jonathan Moules, Florian Gekeler, Andrew R Webster, Susan M Downes, Robert E MacLaren
PURPOSE: Identifying potential biomarkers for disease progression in retinitis pigmentosa (RP) is highly relevant now that gene therapy and other treatments are in clinical trial. Here we report a novel technique for analysis of short-wavelength autofluorescence (AF) imaging to quantify defined regions of AF in RP patients. METHODS: Fifty-five-degree AF images were acquired from 12 participants with RP over a 12-month period. Of these, five were identified as having a hyperfluorescent annulus...
December 2016: Translational Vision Science & Technology
https://www.readbyqxmd.com/read/27932352/local-vascular-gene-therapy-with-apolipoprotein-a-i-to-promote-regression-of-atherosclerosis
#9
Bradley K Wacker, Nagadhara Dronadula, Jingwan Zhang, David A Dichek
OBJECTIVE: Gene therapy, delivered directly to the blood vessel wall, could potentially prevent atherosclerotic lesion growth and promote atherosclerosis regression. Previously, we reported that a helper-dependent adenoviral (HDAd) vector expressing apolipoprotein A-I (apoA-I) in carotid endothelium of fat-fed rabbits reduced early (4 weeks) atherosclerotic lesion growth. Here, we tested whether the same HDAd-delivered to the existing carotid atherosclerotic lesions-could promote regression...
December 8, 2016: Arteriosclerosis, Thrombosis, and Vascular Biology
https://www.readbyqxmd.com/read/27931534/advances-in-clinical-immunology-in-2015
#10
REVIEW
Javier Chinen, Luigi D Notarangelo, William T Shearer
Advances in clinical immunology in the past year included the report of practice parameters for the diagnosis and management of primary immunodeficiencies to guide the clinician in the approach to these relatively uncommon disorders. We have learned of new gene defects causing immunodeficiency and of new phenotypes expanding the spectrum of conditions caused by genetic mutations such as a specific regulator of telomere elongation (RTEL1) mutation causing isolated natural killer cell deficiency and mutations in ras-associated RAB (RAB27) resulting in immunodeficiency without albinism...
December 2016: Journal of Allergy and Clinical Immunology
https://www.readbyqxmd.com/read/27930847/targeting-expression-to-megakaryocytes-and-platelets-by-lineage-specific-lentiviral-vectors
#11
Lisette Johana Latorre-Rey, Sabine Wintterle, Sebastian Dütting, Saskia Kohlscheen, Tobias Abel, Franziska Schenk, Susanne Wingert, Michael A Rieger, Bernhard Nieswandt, Niels Heinz, Ute Modlich
BACKGROUND: Lentiviral transduction and transplantation of hematopoietic stem cells (HSC) can be utilized to modify the phenotype of megakaryocytes and platelets. As the genetic modification in HSC is transmitted onto all hematopoietic progenies, transgene expression from the vector should be restricted to megakaryocytes to avoid un-physiologic effects by ectopic transgene expression. This can be achieved by lentiviral vectors that control expression by lineage-specific promoters. METHODS: in this study, we introduced promoters of megakaryocyte/platelet-specific genes, namely human glycoprotein 6 (hGP6) and hGP9, into third generation lentiviral vectors and analyzed their functionality in-vitro and in-vivo in bone marrow transplantation assays...
December 8, 2016: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/27929686/hematopoietic-cell-transplantation-in-fanconi-anemia-current-evidence-challenges-and-recommendations
#12
Christen L Ebens, Margaret L MacMillan, John E Wagner
Hematopoietic cell transplantation for Fanconi Anemia (FA) has improved dramatically over the past 40 years. With an enhanced understanding of the intrinsic DNA-repair defect and pathophysiology of hematopoietic failure and leukemogenesis, sequential changes to conditioning and graft engineering have significantly improved the expectation of survival after allogeneic hematopoietic cell transplantation (alloHCT) with incidence of graft failure decreased from 35% to <10% and acute graft-versus-host disease (GVHD) from >40% to <10%...
December 8, 2016: Expert Review of Hematology
https://www.readbyqxmd.com/read/27928030/emerging-therapies-for-inherited-retinal-degeneration
#13
REVIEW
Hendrik P N Scholl, Rupert W Strauss, Mandeep S Singh, Deniz Dalkara, Botond Roska, Serge Picaud, José-Alain Sahel
Inherited retinal degenerative diseases, a genetically and phenotypically heterogeneous group of disorders, affect the function of photoreceptor cells and are among the leading causes of blindness. Recent advances in molecular genetics and cell biology are elucidating the pathophysiological mechanisms underlying these disorders and are helping to identify new therapeutic approaches, such as gene therapy, stem cell therapy, and optogenetics. Several of these approaches have entered the clinical phase of development...
December 7, 2016: Science Translational Medicine
https://www.readbyqxmd.com/read/27928011/high-prevalence-of-infectious-adeno-associated-virus-aav-in-human-peripheral-blood-mononuclear-cells-indicative-of-t-lymphocytes-as-sites-of-aav-persistence
#14
Daniela Hüser, Dina Khalid, Timo Lutter, Eva-Maria Hammer, Stefan Weger, Melanie Heßler, Ulrich Kalus, Yvonne Tauchmann, Karin Hensel-Wiegel, Dirk Lassner, Regine Heilbronn
: Seroepidemiology shows that infections with adeno-associated virus (AAV) are widespread, but diverse AAV serotypes isolated from man or non-human primates have so far not been proven as causes of human disease. In view of the increasing success of AAV-derived vectors in human gene therapy, the definition of the in vivo sites of wild-type AAV persistence and clinical consequences of its reactivation are becoming increasingly urgent.Here, we identified the presumed cell-type for AAV persistence in the human host, by highly sensitive AAV PCRs developed for the full spectrum of human AAV serotypes...
December 7, 2016: Journal of Virology
https://www.readbyqxmd.com/read/27927014/genetic-modification-of-the-lung-directed-toward-treatment-of-human-disease
#15
Dolan Sondhi, Katie Stiles, Bishnu P De, Ronald G Crystal
Genetic modification therapy is a promising therapeutic strategy for many diseases of the lung intractable to other treatments. Lung gene therapy has been the subject of numerous preclinical animal experiments and human clinical trials, for targets including genetic diseases such as cystic fibrosis and α1-antitrypsin deficiency, complex disorders such as asthma, allergy and lung cancer, infections such as respiratory syncytial virus and Pseudomonas, as well as pulmonary arterial hypertension, transplant rejection and lung injury...
December 7, 2016: Human Gene Therapy
https://www.readbyqxmd.com/read/27924590/gene-therapy-of-salivary-diseases
#16
Bruce J Baum, Sandra Afione, John A Chiorini, Ana P Cotrim, Corinne M Goldsmith, Changyu Zheng
For many years, our research group worked to develop gene transfer approaches for salivary gland disorders that lacked effective conventional therapy. The purpose of this chapter is to describe and update key methods used in this process. As described in our original chapter from the 2010 volume, we focus on one clinical condition, irradiation-induced salivary hypofunction, and address the choice of transgene and vector to be used, the construction of recombinant viral vectors, how vector delivery is accomplished, and methods for assessing vector function in vitro and in an appropriate animal model...
2017: Methods in Molecular Biology
https://www.readbyqxmd.com/read/27924064/toward-the-use-of-precision-medicine-for-the-treatment-of-head-and-neck-squamous-cell-carcinoma
#17
REVIEW
Wang Gong, Yandi Xiao, Zihao Wei, Yao Yuan, Min Qiu, Chongkui Sun, Xin Zeng, Xinhua Liang, Mingye Feng, Qianming Chen
Precision medicine is a new strategy that aims at preventing and treating human diseases by focusing on individual variations in people's genes, environment and lifestyle. Precision medicine has been used for cancer diagnosis and treatment and shows evident clinical efficacy. Rapid developments in molecular biology, genetics and sequencing technologies, as well as computational technology, has enabled the establishment of "big data", such as the Human Genome Project, which provides a basis for precision medicine...
December 4, 2016: Oncotarget
https://www.readbyqxmd.com/read/27921110/multifunctional-pei-entrapped-gold-nanoparticles-enable-efficient-delivery-of-therapeutic-sirna-into-glioblastoma-cells
#18
Lingdan Kong, Jieru Qiu, Wenjie Sun, Jia Yang, Mingwu Shen, Lu Wang, Xiangyang Shi
RNA interference (RNAi) has been considered as a promising strategy for effective treatment of cancer. However, the easy degradation of small interfering RNA (siRNA) limits its extensive applications in gene therapy. For safe and effective delivery of siRNA, a novel vector system possessing excellent biocompatibility, highly efficient transfection efficiency and specific targeting properties has to be considered. In this study, we report the use of polyethyleneimine (PEI)-entrapped gold nanoparticles (Au PENPs) modified with an arginine-glycine-aspartic (Arg-Gly-Asp, RGD) peptide via a poly(ethylene glycol) (PEG) spacer as a vector for Bcl-2 (B-cell lymphoma-2) siRNA delivery to glioblastoma cells...
December 6, 2016: Biomaterials Science
https://www.readbyqxmd.com/read/27920500/design-synthesis-and-evaluation-of-vegf-sirna-crs-as-a-novel-vector-for-gene-delivery
#19
Wen Zhao, Yifan Zhang, Xueyun Jiang, Chunying Cui
Small interfering RNA (siRNA) delivery is a prospective method in gene therapy, but it has application limitations such as negative charge, water solubility and high molecular weight. In this study, a safe and efficient nano-vector, CRS, was designed and synthesized to facilitate siRNA delivery. Physical and chemical properties of VEGF-siRNA/CRS were characterized by methods including scanning electron microscopy (SEM), transmission electron microscopy, zeta potential (ζ) measurement, drug-releasing rate measurement, gel electrophoresis and confocal microscopy...
2016: Drug Design, Development and Therapy
https://www.readbyqxmd.com/read/27919215/current-strategies-in-modification-of-plga-based-gene-delivery-system
#20
Mohammad Ramezani, Mahboubeh Ebrahimian, Maryam Hashemi
The successful gene therapy has been limited by safe and efficient delivery of nucleic acid to the target cells. Poly (d,l-lactide-co-glycolide) (PLGA) nanoparticles (NPs) are able to deliver drugs and gene efficiently. This formulation has several advantages in comparison with other formulations including improvement of solubility, stability, controlling of degradation and release of the entrapped agents. For application of PLGA as gene carrier, there exist many challenges. PLGA nanoparticles could protect the encapsulated DNA from in vivo degradation but the DNA release is slowl and their negative charge acts as a barrier to DNA incorporation and delivery...
December 5, 2016: Current Medicinal Chemistry
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"