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https://www.readbyqxmd.com/read/28334745/disease-correction-by-aav-mediated-gene-therapy-in-a-new-mouse-model-of-mucopolysaccharidosis-type-iiid
#1
Carles Roca, Sandra Motas, Sara Marcó, Albert Ribera, Víctor Sánchez, Xavier Sánchez, Joan Bertolin, Xavier León, Jennifer Pérez, Miguel Garcia, Pilar Villacampa, Jesús Ruberte, Anna Pujol, Virginia Haurigot, Fatima Bosch
Gene therapy is a promising therapeutic alternative for Lysosomal Storage Disorders (LSD), as it is not necessary to correct the genetic defect in all cells of an organ to achieve therapeutically significant levels of enzyme in body fluids, from which non-transduced cells can uptake the protein correcting their enzymatic deficiency. Animal models are instrumental in the development of new treatments for LSD. Here we report the generation of the first mouse model of the LSD Muccopolysaccharidosis Type IIID (MPSIIID), also known as Sanfilippo syndrome type D...
February 17, 2017: Human Molecular Genetics
https://www.readbyqxmd.com/read/28331098/recombinant-modified-vaccinia-virus-ankara-generating-ebola-virus-like-particles
#2
Marc Schweneker, Andrea S Laimbacher, Gert Zimmer, Susanne Wagner, Elisabeth M Schraner, Michael Wolferstätter, Marieken Klingenberg, Ulrike Dirmeier, Robin Steigerwald, Henning Lauterbach, Hubertus Hochrein, Paul Chaplin, Mark Suter, Jürgen Hausmann
There are currently no approved therapeutics or vaccines to treat or protect against the severe hemorrhagic fever and death caused by Ebola virus (EBOV). Ebola virus-like particles (EBOV-VLPs) consisting of the matrix protein VP40, the glycoprotein (GP) and the nucleoprotein (NP) are highly immunogenic and protective in non-human primates against Ebola virus disease (EVD). We have constructed a modified vaccinia virus Ankara-Bavarian Nordic(®) (MVA-BN(®)) recombinant co-expressing VP40 and glycoprotein (GP) of EBOV Mayinga and the nucleoprotein (NP) of Taï Forest virus (TAFV) (MVA-BN-EBOV-VLP) to launch non-infectious EBOV-VLPs as a second vaccine modality in the MVA-BN-EBOV-VLP-vaccinated organism...
March 22, 2017: Journal of Virology
https://www.readbyqxmd.com/read/28325684/monitoring-and-visualizing-microrna-dynamics-during-live-cell-differentiation-using-microrna-responsive-non-viral-reporter-vectors
#3
Hideyuki Nakanishi, Kenji Miki, Kaoru R Komatsu, Masayuki Umeda, Megumi Mochizuki, Azusa Inagaki, Yoshinori Yoshida, Hirohide Saito
MicroRNA (miRNA) activity differs with cell type, suggesting it can be used as a cell marker. In this study, we developed novel miRNA-responsive non-viral reporter vectors to continuously monitor and visualize miRNA dynamics during differentiation and to efficiently purify target living cells. Each vector codes miRNA-responsive and reference reporter genes in a single mRNA. These two genes are independent modules but transcribed by a single promoter, which enables us to distinguish miRNA-mediated post-transcriptional repression from transcriptional repression...
March 2, 2017: Biomaterials
https://www.readbyqxmd.com/read/28323095/megalin-targeted-enhanced-transfection-efficiency-in-cultured-human-hk-2-renal-tubular-proximal-cells-using-aminoglycoside-carboxyalkyl-polyethylenimine-containing-nanoplexes
#4
Fatemeh Oroojalian, Ali Hossein Rezayan, Wayne Thomas Shier, Khalil Abnous, Mohammad Ramezani
Non-viral vectors are of interest as therapeutic gene delivery agents in gene therapy, because they are simple to prepare, easy to modify and have definable safety profiles compared to viral vectors. The potential of gene therapy in the treatment of renal diseases is limited by a lack of effective kidney-targeted gene delivery systems. Aminoglycoside antibiotics gentamicin and neomycin were connected by amide linkages to carboxyl groups on carboxyalkylated-PEI25 (25kDa PEI) or carboxyalkylated-PEI10 (10kDa PEI)...
March 16, 2017: International Journal of Pharmaceutics
https://www.readbyqxmd.com/read/28322564/bioreducible-zinc-ii-coordinative-pei-with-low-molecular-weight-for-robust-gene-delivery-of-primary-and-stem-cells
#5
Shuai Liu, Dezhong Zhou, Jixiang Yang, Hao Zhou, Jiatong Chen, Tianying Guo
To transform the common low molecular weight (LMW) cationic polymers, such as polyethylenimine (PEI) to highly efficient gene vectors will be of great significance but remains challenging. Since that LMW cationic polymers perform far less efficient to their high molecular weight counterparts mainly due to weaker nucleic acid encapsulation, herein, we report the design and synthesis of a dipicolylamine (DPA)-based disulfide-containing zinc(II) coordinative module (Zn-DDAC), which is used to functionalize LMW PEI (Mw~1800 Da) to give a non-viral vector (Zn-PD) with high efficiency and safety in primary and stem cells...
March 21, 2017: Journal of the American Chemical Society
https://www.readbyqxmd.com/read/28320116/in-vitro-non-viral-murine-pro-neurotrophin-3-gene-transfer-into-rat-bone-marrow-stromal-cells
#6
Shahram Darabi, Taki Tiraihi, AliReza Delshad, Majid Sadeghizadeh, Wisam Khalil, Taher Taheri
Neurotrophin 3 (NT-3) is an important factor for promoting prenatal neural development, as well as regeneration, axogenesis and plasticity in postnatal life. Therapy with NT-3 was reported to improve the condition of patients suffering from degenerative diseases and traumatic injuries, however, the disadvantage of NT-3 protein delivery is its short half-life, thus our alternative approach is the use of NT-3 gene therapy. In this study, the bone marrow stromal cells (BMSCs) were isolated from adult rats, cultured for 4 passages and transfected with either pEGFP-N1 or a constructed vector containing murine proNT-3 (pSecTag2/HygroB-murine proNT-3) using Lipofectamine 2000 followed by Hygromycin B (200mg/kg)...
April 15, 2017: Journal of the Neurological Sciences
https://www.readbyqxmd.com/read/28318036/autophagy-determines-efficiency-of-liver-directed-gene-therapy-with-adeno-associated-viral-vectors
#7
Marianna Hösel, Anke Huber, Susanne Bohlen, Julie Lucifora, Giuseppe Ronzitti, Francesco Puzzo, Florence Boisgerault, Ulrich T Hacker, Wilhelmus J Kwanten, Nora Klöting, Matthias Blüher, Alexander Gluschko, Michael Schramm, Olaf Utermöhlen, Wilhelm Bloch, Federico Mingozzi, Oleg Krut, Hildegard Büning
Use of Adeno-associated viral (AAV) vectors for liver-directed gene therapy has shown considerable success, particularly in patients with severe hemophilia B. However, the high vector doses required to reach therapeutic levels of transgene expression caused liver inflammation in some patients that selectively destroyed transduced hepatocytes. We hypothesized that such detrimental immune responses can be avoided by enhancing the efficacy of AAV vectors in hepatocytes. Because autophagy is a key liver response to environmental stresses, we characterized the impact of hepatic autophagy on AAV infection...
March 20, 2017: Hepatology: Official Journal of the American Association for the Study of Liver Diseases
https://www.readbyqxmd.com/read/28317453/ebola-virus-epidemiology-diagnosis-and-control-threat-to-humans-lessons-learnt-and-preparedness-plans-an-update-on-its-40-year-s-journey
#8
Raj Kumar Singh, Kuldeep Dhama, Yashpal Singh Malik, Muthannan Andavar Ramakrishnan, Kumaragurubaran Karthik, Rekha Khandia, Ruchi Tiwari, Ashok Munjal, Mani Saminathan, Swati Sachan, Perumal Arumugam Desingu, Jobin Jose Kattoor, Hafiz M N Iqbal, Sunil Kumar Joshi
Ebola virus (EBOV) is an extremely contagious pathogen and causes lethal hemorrhagic fever disease in man and animals with high fatality rates. The recently occurred Ebola virus disease (EVD) outbreaks in the West African countries have categorized it as an international health concern. For the virus maintenance and transmission, the non-human primates and reservoir hosts like fruit bats have played a vital role. Furthermore, the virus may also get transferred through contaminated biological fluids like breast milk, tears, urine, semen, blood, etc...
March 20, 2017: Veterinary Quarterly
https://www.readbyqxmd.com/read/28316014/rescue-of-recombinant-newcastle-disease-virus-a-short-history-of-how-it-all-started
#9
REVIEW
Aidin Molouki, Ben Peeters
Reverse genetics of viruses has come a long way, and many recombinant viruses have been generated since the first successful "rescues" were reported in the late 1970s. Recombinant Newcastle disease virus (rNDV), a non-segmented negative-sense RNA virus (NSNSV), was first rescued in 1999 using a reverse genetics approach similar to that reported for other recombinant viruses of the order Mononegavirales a few years before. The route from an original NDV isolate to the generation of its recombinant counterpart requires many steps that have to be sequentially and carefully completed...
March 18, 2017: Archives of Virology
https://www.readbyqxmd.com/read/28315770/biophysics-and-protein-corona-analysis-of-janus-cyclodextrin-dna-nanocomplexes-efficient-cellular-transfection-on-cancer-cells
#10
M Martínez-Negro, G Caracciolo, S Palchetti, D Pozzi, A L Capriotti, C Cavaliere, A Laganà, C Ortiz Mellet, J M Benito, J M García Fernández, E Aicart, E Junquera
The self-assembling processes underlining the capabilities of facially differentiated ("Janus") polycationic amphiphilic cyclodextrins (paCDs) as non-viral gene nanocarriers have been investigated by a pluridisciplinary approach. Three representative Janus paCDs bearing a common tetradecahexanoyl multitail domain at the secondary face and differing in the topology of the cluster of amino groups at the primary side were selected for this study. All of them compact pEGFP-C3 plasmid DNA and promote transfection in HeLa and MCF-7 cells, both in absence and in presence of human serum...
March 15, 2017: Biochimica et Biophysica Acta
https://www.readbyqxmd.com/read/28314126/purification-of-foamy-viral-particles
#11
Ralf Spannaus, Christina Miller, Dirk Lindemann, Jochen Bodem
Foamy viruses are non-pathogenic retroviruses and represent a tool for vector development. For gene therapy applications and for analyses of viral protein composition infectious particles need to be purified, which has been difficult for foamy viruses in the past. Here, we describe a novel, simple, and fast purification method for prototype foamy viruses with high purity using size exclusion and affinity chromatography. More than 99,9% of the contaminating proteins were removed. The purified viruses were used to determine the amount of the incorporated Pol protein relative to Gag...
March 14, 2017: Virology
https://www.readbyqxmd.com/read/28300392/generation-and-characterization-of-virus-infection-enhancing-peptide-nanofibrils-functionalized-with-fluorescent-labels
#12
Sascha Rode, Manuel Hayn, Annika Röcker, Stefanie Sieste, Marcus Lamla, Daniel Markx, Christoph Meier, Frank Kirchhoff, Paul Walther, Marcus Fändrich, Tanja Weil, Jan Muench
Retroviral gene transfer is the method of choice for the stable introduction of genetic material into the cellular genome. However, efficient gene transfer is often limited by low transduction rates of the viral vectors. We have recently described a 12-mer peptide, termed EF-C that forms amyloid-like peptide nanofibrils (PNF) strongly increasing viral transduction efficiencies. These nanofibrils are polycationic and bind negatively charged membranes of virions and cells, thereby overcoming charge repulsions and resulting in increased rates of virion attachment and gene transfer...
March 16, 2017: Bioconjugate Chemistry
https://www.readbyqxmd.com/read/28300083/mr-guided-parenchymal-delivery-of-adeno-associated-viral-vector-serotype-5-in-non-human-primate-brain
#13
L Samaranch, B Blits, W San Sebastian, P Hadaczek, J Bringas, V Sudhakar, M Macayan, P J Pivirotto, H Petry, K S Bankiewicz
The present study was designed to characterize transduction of non-human primate brain and spinal cord with AAV5 viral vector after parenchymal delivery. AAV5-CAG-GFP (1 × 10(13) vector genomes per milliliter (vg ml(-1))) was bilaterally infused either into putamen, thalamus or with the combination left putamen and right thalamus. Robust expression of GFP was seen throughout infusion sites and also in other distal nuclei. Interestingly, thalamic infusion of AAV5 resulted in the transduction of the entire corticospinal axis, indicating transport of AAV5 over long distances...
March 16, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28295042/generation-of-non-viral-transgene-free-hepatocyte-like-cells-with-piggybac-transposon
#14
Hokahiro Katayama, Kentaro Yasuchika, Yuya Miyauchi, Hidenobu Kojima, Ryoya Yamaoka, Takayuki Kawai, Elena Yukie Yoshitoshi, Satoshi Ogiso, Sadahiko Kita, Katsutaro Yasuda, Naoya Sasaki, Ken Fukumitsu, Junji Komori, Takamichi Ishii, Shinji Uemoto
Somatic cells can be reprogrammed to induced hepatocyte-like cells (iHeps) by overexpressing certain defined factors in direct reprogramming techniques. Of the various methods to deliver genes into cells, typically used genome-integrating viral vectors are associated with integration-related adverse events such as mutagenesis, whereas non-integrating viral vectors have low efficiency, making viral vectors unsuitable for clinical application. Therefore, we focused on developing a transposon system to establish a non-viral reprogramming method...
March 15, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28293718/transfection-of-primary-brain-capillary-endothelial-cells-for-protein-synthesis-and-secretion-of-recombinant-erythropoietin-a-strategy-to-enable-protein-delivery-to-the-brain
#15
Annette Burkhart, Thomas Lars Andresen, Achim Aigner, Louiza Bohn Thomsen, Torben Moos
Treatment of chronic disorders affecting the central nervous system (CNS) is complicated by the inability of drugs to cross the blood-brain barrier (BBB). Non-viral gene therapy applied to brain capillary endothelial cells (BCECs) denotes a novel approach to overcome the restraints in this passage, as turning BCECs into recombinant protein factories by transfection could result in protein secretion further into the brain. The present study aims to investigate the possibility of transfecting primary rat brain endothelial cells (RBECs) for recombinant protein synthesis and secretion of the neuroprotective protein erythropoietin (EPO)...
March 14, 2017: Cellular and Molecular Life Sciences: CMLS
https://www.readbyqxmd.com/read/28292248/surface-engineered-dendrimers-in-sirna-delivery-and-gene-silencing
#16
Vishakha Tambe, Shreya Thakkar, Nidhi Raval, Dilip Sharma, Kiran Kalia, Rakesh Kumar Tekade
BACKGROUND: Therapeutic efficacy of dreadful diseases like cancer, HIV (Human Immunodeficiency Virus) can be enhanced by delivering molecules which regulate function at gene level rather than at receptor level. Silencing RNA is one such approach recently used to silence target gene expressed diseases; and thereby reduce target protein levels. Many of the non-viral vectors are proved to act as carriers for silencing RNA. Dendrimers being one of them have less size, low poly dispersibility index, water solubility, multivalence, and easy surface modification...
March 14, 2017: Current Pharmaceutical Design
https://www.readbyqxmd.com/read/28290156/degradable-polyethylenimine-based-gene-carriers-for-cancer-therapy
#17
REVIEW
Hu-Lin Jiang, Mohammad Ariful Islam, Lei Xing, Jannatul Firdous, Wuji Cao, Yu-Jing He, Yong Zhu, Ki-Hyun Cho, Hui-Shan Li, Chong-Su Cho
Gene therapy using recombinant DNA or gene silencing using siRNA have become a prominent area of research in cancer therapy. However, their use in clinical applications is limited due to overall safety concerns and suboptimal efficacy. Although non-viral vectors such as polycationic polymers do not offer the same level of transfection efficiency as their viral counterparts, they still demonstrate immense potential as alternatives to viral vectors, given their versatility, low immunogenicity, ease of large-scale production, and ability to accelerate gene transfer with well-designed delivery platforms...
April 2017: Topics in Current Chemistry (Journal)
https://www.readbyqxmd.com/read/28287517/microrna-based-regulation-of-picornavirus-tropism
#18
Autumn J Ruiz, Stephen J Russell
Cell-specific restriction of viral replication without concomitant attenuation can benefit vaccine development, gene therapy, oncolytic virotherapy, and understanding the biological properties of viruses. There are several mechanisms for regulating viral tropism, however they tend to be virus class specific and many result in virus attenuation. Additionally, many viruses, including picornaviruses, exhibit size constraints that do not allow for incorporation of large amounts of foreign genetic material required for some targeting methods...
February 6, 2017: Journal of Visualized Experiments: JoVE
https://www.readbyqxmd.com/read/28285597/prevalence-study-and-risk-factor-analysis-of-selected-bacterial-protozoal-and-viral-including-vector-borne-pathogens-in-cats-from-cyprus
#19
Charalampos Attipa, Kostas Papasouliotis, Laia Solano-Gallego, Gad Baneth, Yaarit Nachum-Biala, Elpida Sarvani, Toby G Knowles, Sena Mengi, David Morris, Chris Helps, Séverine Tasker
BACKGROUND: Feline infectious agent studies are lacking in Cyprus. The aims of this study were to determine the prevalence and risk factors for various feline infectious agents, including feline vector-borne pathogens (FVBP), in cats from Cyprus. METHODS: A cross-sectional, descriptive, multicentre study was performed on 174 feline samples [138 owned and 36 shelter-feral, including both healthy (43) and non-healthy (131), cats] from private veterinary clinics from all six districts of Cyprus...
March 13, 2017: Parasites & Vectors
https://www.readbyqxmd.com/read/28281871/progress-in-hiv-vaccine-development
#20
Denise C Hsu, Robert J O'Connell
An HIV-1 vaccine is needed to curtail the HIV epidemic. Only one (RV144) out of the 6 HIV-1 vaccine efficacy trials performed showed efficacy. A potential mechanism of protection is the induction of functional antibodies to V1V2 region of HIV envelope. The 2 main current approaches to the generation of protective immunity are through broadly neutralizing antibodies (bnAb) and induction of functional antibodies (non-neutralizing Abs with other potential anti-viral functions). Passive immunization using bnAb has advanced into phase II clinical trials...
March 10, 2017: Human Vaccines & Immunotherapeutics
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