keyword
MENU ▼
Read by QxMD icon Read
search

Lentivirus vector therapy

keyword
https://www.readbyqxmd.com/read/29446741/whats%C3%A2-new-in-gene-therapy-of-hemophilia
#1
E Carlos Rodriguez-Merchan
BACKGROUND: Several methods have been investigated to effectively and safely transmit genes that stimulate cells to release therapeutic factor VIII (FVIII) and factor IX (FIX) into the circulation of people with hemophilia (PWH). OBJECTIVE: To review the role of gene therapy (GT) in PWH. METHODS: A Cochrane Library and PubMed (MEDLINE) search related to the role of GT in hemophilia was analyzed. RESULTS: The most promising vectors for hemophilia GT are adeno-associated virus (AAV) and lentivirus...
February 14, 2018: Current Gene Therapy
https://www.readbyqxmd.com/read/29441951/lncrna-hulc-promotes-epithelial-and-smooth-muscle-like-differentiation-of-adipose-derived-stem-cells-by-upregulation-of-bmp9
#2
Yongwei Li, Zhengfei Shan, Bin Yang, Diandong Yang, Changping Men, Yuanshan Cui, Jitao Wu
AIMS: Adipose-derived stem cells (ADSCs), a source of mesenchymal stem cells, are able to differentiate into numerous cell lineages, including epithelial and smooth muscle cells. The use of ADSCs in tissue engineering technology has become the most promising therapeutic approach for urethral reconstruction. This study aimed to explore the effect of lncRNA highly upregulated in liver cancer (HULC) on the induction of ADSCs to differentiate into epithelial and smooth-muscle-like cells. METHODS: ADSCs were isolated from a male dog, and the expression of HULC in ADSCs was overexpressed by transfection with HULC expressing vector lentivirus...
January 2, 2018: Die Pharmazie
https://www.readbyqxmd.com/read/29409356/therapeutic-potential-of-lentivirus-mediated-glucagon-like-peptide-1-glp-1-gene-therapy-for-diabetes
#3
Hale M Tasyurek, Hasan A Altunbas, Mustafa K Balci, Thomas S Griffith, Salih Sanlioglu
Postprandial glucose-induced insulin secretion from the islets of Langerhans is facilitated by glucagon-like peptide-1 (GLP-1) - a metabolic hormone with insulinotropic properties. Among the variety of effects it mediates, GLP-1 induces delta cell secretion of somatostatin, inhibits alpha cell release of glucagon, reduces gastric emptying, and slows food intake. These events collectively contribute to weight loss over time. During type 2 diabetes (T2DM), however, the incretin response to glucose is reduced and accompanied by a moderate reduction in GLP-1 secretion...
February 6, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29367346/genetically-modified-lentiviruses-that-preserve-microvascular-function-protect-against-late-radiation-damage-in-normal-tissues
#4
Aadil A Khan, James T Paget, Martin McLaughlin, Joan N Kyula, Michelle J Wilkinson, Timothy Pencavel, David Mansfield, Victoria Roulstone, Rohit Seth, Martin Halle, Navita Somaiah, Jessica K R Boult, Simon P Robinson, Hardev S Pandha, Richard G Vile, Alan A Melcher, Paul A Harris, Kevin J Harrington
Improvements in cancer survival mean that long-term toxicities, which contribute to the morbidity of cancer survivorship, are being increasingly recognized. Late adverse effects (LAEs) in normal tissues after radiotherapy (RT) are characterized by vascular dysfunction and fibrosis causing volume loss and tissue contracture, for example, in the free flaps used for immediate breast reconstruction after mastectomy. We evaluated the efficacy of lentivirally delivered superoxide dismutase 2 (SOD2) overexpression and connective tissue growth factor (CTGF) knockdown by short hairpin RNA in reducing the severity of LAEs in an animal model of free flap LAEs...
January 24, 2018: Science Translational Medicine
https://www.readbyqxmd.com/read/29361371/mass-spectrometry-analysis-reveals-differences-in-the-host-cell-protein-species-found-in-pseudotyped-lentiviral-vectors
#5
Sabine Johnson, Jun X Wheeler, Robin Thorpe, Mary Collins, Yasuhiro Takeuchi, Yuan Zhao
Lentiviral vectors (LVs) have been successfully used in clinical trials showing long term therapeutic benefits. Studying the role of cellular proteins in lentivirus HIV-1 life cycle can help understand virus assembly and budding, leading to improvement of LV production for gene therapy. Lentiviral vectors were purified using size exclusion chromatography (SEC). The cellular protein composition of LVs produced by two different methods was compared: the transient transfection system pseudotyped with the VSV-G envelope, currently used in clinical trials, and a stable producer cell system using a non-toxic envelope derived from cat endogenous retrovirus RD114, RDpro...
January 17, 2018: Biologicals: Journal of the International Association of Biological Standardization
https://www.readbyqxmd.com/read/29276180/cd40-knocked-down-tolerogenic-dendritic-cells-decrease-diabetic-injury
#6
Aziz Mahmoudzadeh, Ali Akbar Pourfathollah, Mohammad Hossein Karimi, Seyed Mohammad Moazzeni
BACKGROUND: Type-1 diabetes (T1D) is an autoimmune disease in which T lymphocytes destroy insulin-producing β-cells. Control of self-reactive T lymphocytes and recovery of diabetic injury is the end point of T1D. OBJECTIVE: To investigate generation of tolerogenic dendritic cells (tolDCs) as an innovative method of diabetes therapy. METHODS: Lentivirus vector production was achieved by GIPZ mouse CD40 shRNA, psPAX2 and pMD2G plasmids DNA...
December 2017: Iranian Journal of Immunology: IJI
https://www.readbyqxmd.com/read/29258934/lentivirus-is-an-efficient-and-stable-transduction-vector-for-intervertebral-disc-cells
#7
Yue-Hui Zhang, Yang-Lu Zhao, Bo Li, Jia Song, Jing Zhang, Jiang Shao
OBJECTIVES: To evaluate the transduction efficacy and sustainability of lentiviral vector for intervertebral disc cells, both in vitro and in vivo. METHORDS: Human nuclear pulposus and annulus fibrosus cells isolated from disc tissue of 28 patients during surgical disc procedures were cultured, and subsequently transduced using recombinant lentivirus carrying a gene for enhanced green fluorescent protein (EGFP) at multiplicities of infection (MOI) of 0, 15, 30, 60, 90, and 150...
December 16, 2017: World Neurosurgery
https://www.readbyqxmd.com/read/29246327/in%C3%A2-vivo-knockout-of-the-vegfa-gene-by-lentiviral-delivery-of-crispr-cas9-in-mouse-retinal-pigment-epithelium-cells
#8
Andreas Holmgaard, Anne Louise Askou, Josephine Natalia Esther Benckendorff, Emil Aagaard Thomsen, Yujia Cai, Toke Bek, Jacob Giehm Mikkelsen, Thomas J Corydon
Virus-based gene therapy by CRISPR/Cas9-mediated genome editing and knockout may provide a new option for treatment of inherited and acquired ocular diseases of the retina. In support of this notion, we show that Streptococcus pyogenes (Sp) Cas9, delivered by lentiviral vectors (LVs), can be used in vivo to selectively ablate the vascular endothelial growth factor A (Vegfa) gene in mice. By generating LVs encoding SpCas9 targeted to Vegfa, and in parallel the fluorescent eGFP marker protein, we demonstrate robust knockout of Vegfa that leads to a significant reduction of VEGFA protein in transduced cells...
December 15, 2017: Molecular Therapy. Nucleic Acids
https://www.readbyqxmd.com/read/29233896/retargeting-lentiviruses-via-spycatcher-spytag-chemistry-for-gene-delivery-into-specific-cell-types
#9
Nagarjun Kasaraneni, Ana M Chamoun-Emanuelli, Gus Wright, Zhilei Chen
We report a simple strategy for the creation of lentiviral vectors specific to any desired target cells. SpyTag is inserted into an engineered Sindbis virus envelope protein and displayed on the lentivirus surface to create Sindbis virus-SpyTag pseudoparticles (Sind-SpyTag-pp). The SpyTag serves as the covalent anchoring site for a target-cell-specific cell-binding protein (CBP) that is fused to a truncated SpyCatcher (SpyCatcherΔ). Target-cell-specific lentiviruses are created by mixing the Sind-SpyTag-pp and CBP-SpyCatcherΔ in vitro We first used a HER2-binding designed ankyrin repeat protein (DARPin...
December 12, 2017: MBio
https://www.readbyqxmd.com/read/29221804/the-impact-of-the-cd9-tetraspanin-on-lentivirus-infectivity-and-exosome-secretion
#10
Kai O Böker, Nicolas Lemus-Diaz, Rafael Rinaldi Ferreira, Lara Schiller, Stefan Schneider, Jens Gruber
Efficient transduction tools are a hallmark for both research and therapy development. Here, we introduce new insights into the generation of lentiviral vectors with improved performance by utilizing producer cells with increased production rates of extracellular vesicles through CD9 overexpression. Most human cells secrete small vesicles from their surface (microvesicles) or intraluminal endosome-derived membranes (exosomes). In particular, enhanced levels of the tetraspanin CD9 result in significantly increased numbers of extracellular vesicles with exosome-like features that were secreted from four different human cell lines...
November 16, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29212357/manufacture-of-third-generation-lentivirus-for-preclinical-use-with-process-development-considerations-for-translation-to-good-manufacturing-practice-gmp
#11
Carolina Gándara, Valerie Affleck, Elizabeth Ann Stoll
Lentiviral vectors are used in laboratories around the world for in vivo and ex vivo delivery of gene therapies, and increasingly used for clinical investigation as well as preclinical applications. The third-generation lentiviral vector system has many advantages, including high packaging capacity, stable gene expression in both dividing and post-mitotic cells, and low immunogenicity in the recipient organism. Yet the manufacture of these vectors is challenging, especially at high titres required for direct use in vivo, and further challenges are presented by the process of translating preclinical gene therapies toward manufacture of products for clinical investigation...
December 6, 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/29203150/retroviral-and-lentiviral-safety-analysis-of-gene-modified-t-cell-products-and-infused-hiv-and-oncology-patients
#12
Katherine T Marcucci, Julie K Jadlowsky, Wei-Ting Hwang, Megan Suhoski-Davis, Vanessa E Gonzalez, Irina Kulikovskaya, Minnal Gupta, Simon F Lacey, Gabriela Plesa, Anne Chew, J Joseph Melenhorst, Bruce L Levine, Carl H June
Replication-competent retrovirus/lentivirus (RCR/L) and insertional oncogenesis are potential safety risks with integrating viruses in gene-modified cell therapies. As such, the Food and Drug Administration guidances outline RCR/L-monitoring methods throughout the entire gene therapy treatment cycle. We present data for 17 vector lots, 375 manufactured T cell products, and 308 patients post-infusion across both HIV and oncology indications, showing no evidence of RCR/L. Given our data, a Poisson probability model estimates that a single patient, or a group of patients, would need to be followed for at least 52...
October 20, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29188505/development-of-multigenic-lentiviral-vectors-for-cell-specific-expression-of-antiangiogenic-mirnas-and-protein-factors
#13
Anne Louise Askou, Thomas J Corydon
Generation of lentivirus (LV)-based vectors holding multiple gene cassettes for coexpression of several therapeutic factors provides potent tools in both gene delivery studies as well as in gene therapy. Here we describe the development of such multigenic LV gene delivery vectors enabling cell-specific coexpression of antiangiogenic microRNA (miRNA) and protein factors and, if preferred, a fluorescent reporter, from RNApol(II)-driven expression cassettes orientated in a back-to-back fashion. This configuration may contribute to the development of new combination therapies for amelioration of diseases involving intraocular neovascularization including exudative age-related macular degeneration (AMD)...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29179571/the-role-of-basal-cells-in-producing-persistent-lentivirus-mediated-airway-gene-expression
#14
Nigel R Farrow, Martin Donnelley, Patricia Cmielewski, Eugene Roscioli, Nathan Rout-Pitt, Chantelle McIntyre, Ivan Bertoncello, David Parsons
RATIONALE: Cystic Fibrosis (CF) lung disease is an ideal candidate for a genetic therapy. We have previously shown that pre-conditioning with lysophosphatidylcholine (LPC) prior to lentiviral (LV) vector delivery results in long-term in vivo gene expression in the airway epithelium of CF mice. We hypothesise that this outcome is largely due to transduction of airway basal cells that in turn pass the transgene onto their progeny. OBJECTIVES: The aim of these studies was to confirm if the in vivo delivery of a HIV-1 VSV-G pseudotyped LV vector following LPC airway conditioning results in transduction of mouse airway basal cells in situ and if the transgene is passed onto their progeny...
November 27, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/29159200/analyzing-the-genotoxicity-of-retroviral-vectors-in-hematopoietic-cell-gene-therapy
#15
REVIEW
Luca Biasco, Michael Rothe, Hildegard Büning, Axel Schambach
Retroviral vectors, including those derived from gammaretroviruses and lentiviruses, have found their way into the clinical arena and demonstrated remarkable efficacy for the treatment of immunodeficiencies, leukodystrophies, and globinopathies. Despite these successes, gene therapy unfortunately also has had to face severe adverse events in the form of leukemias and myelodysplastic syndromes, related to the semi-random vector integration into the host cell genome that caused deregulation of neighboring proto-oncogenes...
March 16, 2018: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/29150004/dynamics-of-indel-profiles-induced-by-various-crispr-cas9-delivery-methods
#16
Michael Kosicki, Sandeep S Rajan, Flaminia C Lorenzetti, Hans H Wandall, Yoshiki Narimatsu, Emmanouil Metzakopian, Eric P Bennett
The introduction of CRISPR/Cas9 gene editing in mammalian cells is a scientific breakthrough, which has greatly affected basic research and gene therapy. The simplicity and general access to CRISPR/Cas9 reagents has in an unprecedented manner "democratized" gene targeting in biomedical research, enabling genetic engineering of any gene in any cell, tissue, organ, and organism. The ability for fast, precise, and efficient profiling of the double-stranded break induced insertions and deletions (indels), mediated by any of the available programmable nucleases, is paramount to any given gene targeting approach...
2017: Progress in Molecular Biology and Translational Science
https://www.readbyqxmd.com/read/29130351/the-gene-therapy-resource-program-a-decade-of-dedication-to-translational-research-by-the-national-heart-lung-and-blood-institute
#17
Terence R Flotte, Eric Daniels, Janet Benson, Jeneé M Bevett-Rose, Kenneth Cornetta, Margaret Diggins, Julie Johnston, Susan Sepelak, Johannes C M van der Loo, James M Wilson, Cheryl L McDonald
Over a 10-year period, the Gene Therapy Resource Program (GTRP) of the National Heart Lung and Blood Institute has provided a set of core services to investigators to facilitate the clinical translation of gene therapy. These services have included a preclinical (research-grade) vector production core; current Good Manufacturing Practice clinical-grade vector cores for recombinant adeno-associated virus and lentivirus vectors; a pharmacology and toxicology core; and a coordinating center to manage program logistics and to provide regulatory and financial support to early-phase clinical trials...
December 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/29105567/in-vivo-survival-of-human-endometrial-mesenchymal-stem-cells-transplanted-under-the-kidney-capsule-of-immunocompromised-mice
#18
Shanti Gurung, James A Deane, Saeedeh Darzi, Jerome A Werkmeister, Caroline E Gargett
Human endometrial mesenchymal stem cells (eMSCs) are a well-characterised adult stem cell type with potential for use in regenerative medicine or cell-therapy. As a proof of principle, we demonstrated that eMSCs promoted wound healing by reducing the inflammatory response through a paracrine action in a subcutaneous rat model of wound repair. However, an efficient protocol for culturing eMSCs in the undifferentiated state and a reliable method of labelling them for cell tracking were lacking. Here, we investigated the use of a lentiviral vector containing the mCherry fluorescent reporter gene to transduce and label eMSCs following in vitro culturing in A83-01-containing medium, and different methods of tracing the labelled cells following transplantation under the kidney capsule of immunocompromised NSG mice...
November 4, 2017: Stem Cells and Development
https://www.readbyqxmd.com/read/29077949/incorporation-of-aptamers-in-the-terminal-loop-of-shrnas-yields-an-effective-and-novel-combinatorial-targeting-strategy
#19
Ka Ming Pang, Daniela Castanotto, Haitang Li, Lisa Scherer, John J Rossi
Gene therapy by engineering patient's own blood cells to confer HIV resistance can potentially lead to a functional cure for AIDS. Toward this goal, we have previously developed an anti-HIV lentivirus vector that deploys a combination of shRNA, ribozyme and RNA decoy. To further improve this therapeutic vector against viral escape, we sought an additional reagent to target HIV integrase. Here, we report the development of a new strategy for selection and expression of aptamer for gene therapy. We developed a SELEX protocol (multi-tag SELEX) for selecting RNA aptamers against proteins with low solubility or stability, such as integrase...
January 9, 2018: Nucleic Acids Research
https://www.readbyqxmd.com/read/29057663/gene-therapy-for-inherited-retinal-and-optic-nerve-degenerations
#20
Nicholas A Moore, Nuria Morral, Thomas A Ciulla, Peter Bracha
The eye is a target for investigational gene therapy due to the monogenic nature of many inherited retinal and optic nerve degenerations (IRD), its accessibility, tight blood-ocular barrier, the ability to non-invasively monitor for functional and anatomic outcomes, as well as its relative immune privileged state.Vectors currently used in IRD clinical trials include adeno-associated virus (AAV), small single-stranded DNA viruses, and lentivirus, RNA viruses of the retrovirus family. Both can transduce non-dividing cells, but AAV are non-integrating, while lentivirus integrate into the host cell genome, and have a larger transgene capacity...
October 23, 2017: Expert Opinion on Biological Therapy
keyword
keyword
43309
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"