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https://www.readbyqxmd.com/read/29236688/evolution-of-a-designed-protein-assembly-encapsulating-its-own-rna-genome
#1
Gabriel L Butterfield, Marc J Lajoie, Heather H Gustafson, Drew L Sellers, Una Nattermann, Daniel Ellis, Jacob B Bale, Sharon Ke, Garreck H Lenz, Angelica Yehdego, Rashmi Ravichandran, Suzie H Pun, Neil P King, David Baker
The challenges of evolution in a complex biochemical environment, coupling genotype to phenotype and protecting the genetic material, are solved elegantly in biological systems by the encapsulation of nucleic acids. In the simplest examples, viruses use capsids to surround their genomes. Although these naturally occurring systems have been modified to change their tropism and to display proteins or peptides, billions of years of evolution have favoured efficiency at the expense of modularity, making viral capsids difficult to engineer...
December 13, 2017: Nature
https://www.readbyqxmd.com/read/29229433/establishment-of-integration-free-induced-pluripotent-stem-cells-from-human-recessive-dystrophic-epidermolysis-bullosa-keratinocytes
#2
Wakana Matsumura, Yasuyuki Fujita, Chihiro Nakayama, Satoru Shinkuma, Shotaro Suzuki, Toshifumi Nomura, Riichiro Abe, Hiroshi Shimizu
BACKGROUND: Induced pluripotent stem cell (iPSC) technology enables patient-specific pluripotent stem cells to be derived from adult somatic cells without the use of an embryonic cell source. To date, recessive dystrophic epidermolysis bullosa (RDEB)-specific iPSCs have been generated from patients using integrating retroviral vectors. However, vector integration into the host genome can endanger the biosafety and differentiation propensities of iPSCs. Although various integration-free reprogramming systems have been reported, their utility in reprogramming somatic cells from patients remains largely undetermined...
December 1, 2017: Journal of Dermatological Science
https://www.readbyqxmd.com/read/29221807/ex%C3%A2-vivo-selection-of-transduced-hematopoietic-stem-cells-for-gene-therapy-of-%C3%AE-hemoglobinopathies
#3
Kanit Bhukhai, Edouard de Dreuzy, Marie Giorgi, Charlotte Colomb, Olivier Negre, Maria Denaro, Béatrix Gillet-Legrand, Joëlle Cheuzeville, Anaïs Paulard, Hélène Trebeden-Negre, Suparerk Borwornpinyo, Karine Sii-Felice, Leila Maouche, Julian D Down, Phillippe Leboulch, Emmanuel Payen
Although gene transfer to hematopoietic stem cells (HSCs) has shown therapeutic efficacy in recent trials for several individuals with inherited disorders, transduction incompleteness of the HSC population remains a hurdle to yield a cure for all patients with reasonably low integrated vector numbers. In previous attempts at HSC selection, massive loss of transduced HSCs, contamination with non-transduced cells, or lack of applicability to large cell populations has rendered the procedures out of reach for human applications...
October 24, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29213085/effective-in-vivo-gene-delivery-with-reduced-toxicity-achieved-by-charge-and-fatty-acid-modified-cell-penetrating-peptide
#4
Kaido Kurrikoff, Kadi-Liis Veiman, Kadri Künnapuu, Elin Madli Peets, Tõnis Lehto, Ly Pärnaste, Piret Arukuusk, Ülo Langel
Non-viral gene delivery systems have gained considerable attention as a promising alternative to viral delivery to treat diseases associated with aberrant gene expression. However, regardless of extensive research, only a little is known about the parameters that underline in vivo use of the nanoparticle-based delivery vectors. The modest efficacy and low safety of non-viral delivery are the two central issues that need to be addressed. We have previously characterized an efficient cell penetrating peptide, PF14, for in vivo applications...
December 6, 2017: Scientific Reports
https://www.readbyqxmd.com/read/29204900/molecular-design-for-recombinant-adeno-associated-virus-raav-vector-production
#5
REVIEW
Juan Jose Aponte-Ubillus, Daniel Barajas, Joseph Peltier, Cameron Bardliving, Parviz Shamlou, Daniel Gold
Recombinant adeno-associated virus (rAAV) vectors are increasingly popular tools for gene therapy applications. Their non-pathogenic status, low inflammatory potential, availability of viral serotypes with different tissue tropisms, and prospective long-lasting gene expression are important attributes that make rAAVs safe and efficient therapeutic options. Over the last three decades, several groups have engineered recombinant AAV-producing platforms, yielding high titers of transducing vector particles. Current specific productivity yields from different platforms range from 103 to 105 vector genomes (vg) per cell, and there is an ongoing effort to improve vector yields in order to satisfy high product demands required for clinical trials and future commercialization...
December 4, 2017: Applied Microbiology and Biotechnology
https://www.readbyqxmd.com/read/29204184/viral-cre-loxp-tools-aid-genome-engineering-in-mammalian-cells
#6
Ranjita Sengupta, Amy Mendenhall, Nandita Sarkar, Chandreyee Mukherjee, Amirali Afshari, Joseph Huang, Biao Lu
Background: Targeted nucleases have transformed genome editing technology, providing more efficient methods to make targeted changes in mammalian genome. In parallel, there is an increasing demand of Cre-LoxP technology for complex genome manipulation such as large deletion, addition, gene fusion and conditional removal of gene sequences at the target site. However, an efficient and easy-to-use Cre-recombinase delivery system remains lacking. Results: We designed and constructed two sets of expression vectors for Cre-recombinase using two highly efficient viral systems, the integrative lentivirus and non-integrative adeno associated virus...
2017: Journal of Biological Engineering
https://www.readbyqxmd.com/read/29197187/development-of-a-microfluidic-platform-for-high-throughput-screening-of-non-viral-gene-delivery-vectors
#7
Elisa Giupponi, Roberta Visone, Paola Occhetta, Federica Colombo, Marco Rasponi, Gabriele Candiani
The grail of gene delivery is the development of delivery vectors as effective and non-cytotoxic as possible. In this regard, there is an urgent need of new tools for the straightforward and quantitative assessment of transfection efficiency and cytotoxicity simultaneously. We herein reported the development and validation of an easy-to-use lab-on-chip platform to perform cell transfection assays for unbiased, high-throughput selection of more and more effective gene delivery vectors by using 2 commercially sourced lipids, Lipofectamine 2000® and FuGene® 6...
December 2, 2017: Biotechnology and Bioengineering
https://www.readbyqxmd.com/read/29192808/a-splice-site-variant-in-flvcr1-produces-retinitis-pigmentosa-without-posterior-column-ataxia
#8
Imran H Yusuf, Morag E Shanks, Penny Clouston, Robert E MacLaren
FLVCR1 (feline leukemia virus subgroup c receptor 1) is a transmembrane protein involved in the trafficking of intracellular heme. Homozygous variants in FLVCR1 have been described in association with a clinical syndrome of posterior column ataxia with retinitis pigmentosa (PCARP). Here, we describe a patient with non-syndromic retinitis pigmentosa homozygous for a splice-site variant in FLVCR1 (c.1092 + 5G>A) without evidence of posterior column ataxia or cerebellar degeneration. We suggest an association between intronic splice-site variants in FLVCR1 and the absence of posterior column degeneration and suggest a hypothesis to explain this observation...
December 1, 2017: Ophthalmic Genetics
https://www.readbyqxmd.com/read/29191424/receptor-mediated-targeted-delivery-of-dna-using-lactoferrin-nanoparticles
#9
Sonali Kumari, Anand Kumar Kondapi
Efficient gene delivery facilitated by non-viral vectors, is comparatively safer alternative than viral carriers. Current approaches to gene delivery largely depends on methods that overcome cellular and tissue barriers impeding efficient DNA delivery. While the conventional delivery systems have the drawback of low cellular uptake and off target effects, the receptor-mediated gene delivery system has shown remarkable breakthrough. In that context exploiting the specific receptor targeting properties of lactoferrin protein, we have developed pDNA loaded lactoferrin protein nanoparticles (pDNA-LfNPs)...
November 27, 2017: International Journal of Biological Macromolecules
https://www.readbyqxmd.com/read/29190455/structural-features-of-the-salivary-gland-hypertrophy-virus-of-the-tsetse-fly-revealed-by-cryo-electron-microscopy-and-tomography
#10
Igor Orlov, Robert Drillien, Danièle Spehner, Max Bergoin, Adly M M Abd-Alla, Bruno P Klaholz
Glossina palipides salivary gland hypertrophy virus (GpSGHV) infects tsetse flies, which are vectors for African trypanosomosis. This virus represents a major challenge in insect mass rearing and has hampered the implementation of the sterile insect technique programs in the Member States of the International Atomic Energy Agency. GpSGHV virions consist of long rod-shaped particles over 9000Å in length, but little is known about their detailed structural organization. We show by cryo electron microscopy and cryo electron tomography that the GpSGHV virion has a unique, non-icosahedral helical structure...
November 27, 2017: Virology
https://www.readbyqxmd.com/read/29175632/intravenous-administration-of-the-adeno-associated-virus-php-b-capsid-fails-to-upregulate-transduction-efficiency-in-the-marmoset-brain
#11
Yasunori Matsuzaki, Ayumu Konno, Ryuta Mochizuki, Yoichiro Shinohara, Keisuke Nitta, Yukihiro Okada, Hirokazu Hirai
Intravenous administration of adeno-associated virus (AAV)-PHP.B, a capsid variant of AAV9 containing seven amino acid insertions, results in a greater permeability of the blood brain barrier (BBB) than standard AAV9 in mice, leading to highly efficient and global transduction of the central nervous system (CNS). The present study aimed to examine whether the enhanced BBB penetrance of AAV-PHP.B observed in mice also occurs in non-human primates. Thus, a young adult (age, 1.6 years) and an old adult (age, 7...
November 23, 2017: Neuroscience Letters
https://www.readbyqxmd.com/read/29175597/delivery-of-nf-%C3%AE%C2%BAb-shrna-using-carbamate-mannose-modified-pei-for-eliminating-cancer-stem-cells
#12
Xiyu Ke, Chuan Yang, Wei Cheng, Yi Yan Yang
The presence of cancer stem cells (CSCs) is one of the main reasons that cause cancer relapse and metastasis. In this study, NF-κB shRNA was delivered to target CSCs using carbamate-mannose modified PEI (CMP) as a non-viral gene vector. The polymer was synthesized by blocking primary amine groups of branched PEI (10kDa) through nucleophilic addition between PEI and protected mannose-functionalized cyclic carbonate, followed by mannose deprotection. CMP/control shRNA nanocomplexes showed lower cytotoxicity and higher transfection efficiency in 4 T1 murine breast cancer cells than unmodified PEI/control shRNA nanocomplexes...
November 22, 2017: Nanomedicine: Nanotechnology, Biology, and Medicine
https://www.readbyqxmd.com/read/29175439/lipid-coated-chitosan-dna-nanoparticles-for-enhanced-gene-delivery
#13
Elias Baghdan, Shashank Reddy Pinnapireddy, Boris Strehlow, Konrad H Engelhardt, Jens Schäfer, Udo Bakowsky
Chitosan as a polycationic non-viral vector for gene delivery has the advantage of being a biocompatible and biodegradable polymer. However, without laborious chemical modifications to its structure, it is of limited use as a gene delivery vehicle due to its low ability to efficiently transfect under physiological conditions. To address this problem, we developed novel liposome encapsulated chitosan nanoparticles; lipochitoplexes (LCPs). Chitosan nanoparticles (CsNPs) were obtained using the ionic gelation technique...
November 22, 2017: International Journal of Pharmaceutics
https://www.readbyqxmd.com/read/29175033/evidence-of-varroa-mediated-deformed-wing-virus-spillover-in-hawaii
#14
Jessika Santamaria, Ethel M Villalobos, Laura E Brettell, Scott Nikaido, Jason R Graham, Stephen Martin
Varroa destructor, a parasitic mite of honey bees, is also a vector for viral diseases. The mite displays high host specificity and requires access to colonies of Apis spp. to complete its lifecycle. In contrast, the Deformed Wing Virus (DWV), one of the many viruses transmitted by V. destructor, appears to have a much broader host range. Previous studies have detected DWV in a variety of insect groups that are not directly parasitized by the mite. In this study, we take advantage of the discrete distribution of the Varroa mite in the Hawaiian archipelago to compare DWV prevalence on non-Apis flower visitors, and test whether Varroa presence is linked to a "viral spillover"...
November 21, 2017: Journal of Invertebrate Pathology
https://www.readbyqxmd.com/read/29164070/generation-of-an-attenuated-tiantan-vaccinia-virus-strain-by-deletion-of-multiple-genes
#15
Yiquan Li, Yilong Zhu, Shuang Chen, Wenjie Li, Xunzhe Yin, Shanzhi Li, Pengpeng Xiao, Jicheng Han, Xiao Li, Lili Sun, Ningyi Jin
An attenuated vaccinia virus-MVTTEAB-was constructed by deletion of non-essential gene segments related to the immunomodulatory and virulence functions of the vaccinia virus Tiantan strain (VVTT). The shuttle plasmids pTC-EGFP, pTE-EGFP, pTA35-EGFP, pTB-EGFP, and pTA66-EGFP were constructed and combined with the early and late strong promoter pE/L and EGFP as an exogenous selectable marker. Then, through the homologous recombination technology and Cre/loxP system, the following gene fragments were gradually knocked out one by one: TC7L-TK2L, TE3L, TA35R, TB13R, and TA66R...
2017: Frontiers in Cellular and Infection Microbiology
https://www.readbyqxmd.com/read/29163561/expression-of-separate-proteins-in-the-same-plant-leaves-and-cells-using-two-independent-virus-based-gene-vectors
#16
Maria R Mendoza, Alexandria N Payne, Sean Castillo, Megan Crocker, Brian D Shaw, Herman B Scholthof
Plant viral vectors enable the expression of proteins at high levels in a relatively short time. For many purposes (e.g., cell biological interaction studies) it may be desirable to express more than one protein in a single cell but that is often not feasible when using a single virus vector. Such a co-expression strategy requires the simultaneous delivery by two compatible and non-competitive viruses that can co-exist to each express a separate protein. Here, we report on the use of two agro-launchable coat-protein gene substitution GFP-expressing virus vector systems based on Tomato bushy stunt virus (TBSV) referred to as TG, and Tobacco mosaic virus (TMV) annotated as TRBO-G...
2017: Frontiers in Plant Science
https://www.readbyqxmd.com/read/29160221/evaluation-of-blue-mussel-mytilus-edulis-as-vector-for-viral-hemorrhagic-septicemia-virus-vhsv
#17
Kwang Il Kim, Young Chul Kim, Woo Ju Kwon, Hyun Do Jeong
When viral diseases occur in aquaculture farms, the virus released into the seawater from infected animals can re-infect other susceptible species or accumulate in filter-feeding organisms. We conducted a viral hemorrhagic septicemia virus (VHSV) survivability analysis of blue mussel Mytilus edulis digestive enzymes, viral depuration, and infectivity tests via in vitro and in vivo inoculation to evaluate the infectious state. VHSV particles were not completely digested within 24 h in vitro and were maintained for 7 d in the mussel digestive gland...
November 21, 2017: Diseases of Aquatic Organisms
https://www.readbyqxmd.com/read/29151352/plasmid-dna-delivery-nanotopography-matters
#18
Hao Song, Meihua Yu, Yao Lu, Zhengying Gu, Yannan Yang, Min Zhang, Jianye Fu, Chengzhong Yu
Plasmid DNA molecules with unique loop structures have widespread bio-applications, in many cases relying heavily on delivery vehicles to introduce them into cells and achieve their functions. Herein, we demonstrate that control over delicate nanotopography of silica nanoparticles as plasmid DNA vectors has significant impact on the transfection efficacy. For silica nanoparticles with rambutan-, raspberry- and flower-like morphologies comprised of spike, hemisphere and bowl type subunit nanotopographies, respectively, the rambutan-like nanoparticles with spiky surfaces demonstrate the highest plasmid DNA binding capability and transfection efficacy of 88%, higher than reported silica based nano-vectors...
November 20, 2017: Journal of the American Chemical Society
https://www.readbyqxmd.com/read/29149775/lentiviral-vectors-and-adeno-associated-virus-vectors-useful-tools-for-gene-transfer-in-pain-research
#19
REVIEW
Zheng Chen-Xi, Wang Sheng-Ming, Bai Yun-Hu, Luo Ting-Ting, Wang Jia-Qi, Dai Chun-Qiu, Guo Bao-Lin, Luo Shi-Cheng, Wang Dong-Hui, Yang Yan-Ling, Wang Ya-Yun
Pain, especially chronic pain, has always been a heated point in both basic and clinical researches since it puts heavy burdens on both individuals and the whole society. A better understanding of the role of biological molecules and various ionic channels involved in pain can shed light on the mechanism under pain and advocate the development of pain management. Using viral vectors to transfer specific genes at targeted sites is a promising method for both research and clinical applications. Lentiviral vectors and adeno-associated virus (AAV) vectors which allow stable and long-term expression of transgene in non-dividing cells are widely applied in pain research...
November 17, 2017: Anatomical Record: Advances in Integrative Anatomy and Evolutionary Biology
https://www.readbyqxmd.com/read/29145481/amiloride-enhanced-gene-transfection-of-octa-arginine-functionalized-calcium-phosphate-nanoparticles
#20
Juan Ramón Vanegas Sáenz, Taichi Tenkumo, Yuya Kamano, Hiroshi Egusa, Keiichi Sasaki
Nanoparticles represent promising gene delivery systems in biomedicine to facilitate prolonged gene expression with low toxicity compared to viral vectors. Specifically, nanoparticles of calcium phosphate (nCaP), the main inorganic component of human bone, exhibit high biocompatibility and good biodegradability and have been reported to have high affinity for protein or DNA, having thus been used as gene transfer vectors. On the other hand, Octa-arginine (R8), which has a high permeability to cell membrane, has been reported to improve intracellular delivery systems...
2017: PloS One
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