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Lentivirus vector therapy

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https://www.readbyqxmd.com/read/27891535/gene-therapy-for-hemoglobin-disorders-a-mini-review
#1
Parul Rai, Punam Malik
Gene therapy by either gene insertion or editing is an exciting curative therapeutic option for monogenic hemoglobin disorders like sickle cell disease and β-thalassemia. The safety and efficacy of gene transfer techniques has markedly improved with the use of lentivirus vectors. The clinical translation of this technology has met with good success, although key limitations include number of engraftable transduced hematopoietic stem cells and adequate transgene expression that results in complete correction of β0 thalassemia major...
2016: Journal of Rare Diseases Research & Treatment
https://www.readbyqxmd.com/read/27889981/chimeric-trojan-protein-insertion-in-lentiviral-membranes-makes-lentiviruses-susceptible-to-neutralisation-by-anti-tetanus-serum-antibodies
#2
Anita Le-Heron, Steve Patterson, Rafael J Yáñez-Muñoz, George Dickson
This study describes the initial testing of a novel strategy for neutralisation of lentiviruses using the fundamental biology of enveloped viruses' assembly and budding. In the field of gene therapy, viral vector surface proteins have been manipulated in order to redirect host cell specificity by alteration of pseudo-types. We tested whether known viral pseudo-typing proteins or surface proteins known to be recruited to the HIV envelope could be engineered to carry neutralising epitopes from another microorganism onto the lentiviral surface...
November 27, 2016: Human Gene Therapy
https://www.readbyqxmd.com/read/27857058/molecular-imaging-and-cancer-gene-therapy
#3
REVIEW
Z Saadatpour, G Bjorklund, S Chirumbolo, M Alimohammadi, H Ehsani, H Ebrahiminejad, H Pourghadamyari, B Baghaei, H R Mirzaei, A Sahebkar, H Mirzaei, M Keshavarzi
Gene therapy is known as one of the most advanced approaches for therapeutic prospects ranging from tackling genetic diseases to combating cancer. In this approach, different viral and nonviral vector systems such as retrovirus, lentivirus, plasmid and transposon have been designed and employed. These vector systems are designed to target different therapeutic genes in various tissues and cells such as tumor cells. Therefore, detection of the vectors containing therapeutic genes and monitoring of response to the treatment are the main issues that are commonly faced by researchers...
November 18, 2016: Cancer Gene Therapy
https://www.readbyqxmd.com/read/27852956/preparation-for-a-first-in-man-lentivirus-trial-in-patients-with-cystic-fibrosis
#4
Eric W F W Alton, Jeffery M Beekman, A Christopher Boyd, June Brand, Marianne S Carlon, Mary M Connolly, Mario Chan, Sinead Conlon, Heather E Davidson, Jane C Davies, Lee A Davies, Johanna F Dekkers, Ann Doherty, Sabrina Gea-Sorli, Deborah R Gill, Uta Griesenbach, Mamoru Hasegawa, Tracy E Higgins, Takashi Hironaka, Laura Hyndman, Gerry McLachlan, Makoto Inoue, Stephen C Hyde, J Alastair Innes, Toby M Maher, Caroline Moran, Cuixiang Meng, Michael C Paul-Smith, Ian A Pringle, Kamila M Pytel, Andrea Rodriguez-Martinez, Alexander C Schmidt, Barbara J Stevenson, Stephanie G Sumner-Jones, Richard Toshner, Shu Tsugumine, Marguerite W Wasowicz, Jie Zhu
We have recently shown that non-viral gene therapy can stabilise the decline of lung function in patients with cystic fibrosis (CF). However, the effect was modest, and more potent gene transfer agents are still required. Fuson protein (F)/Hemagglutinin/Neuraminidase protein (HN)-pseudotyped lentiviral vectors are more efficient for lung gene transfer than non-viral vectors in preclinical models. In preparation for a first-in-man CF trial using the lentiviral vector, we have undertaken key translational preclinical studies...
November 16, 2016: Thorax
https://www.readbyqxmd.com/read/27769802/challenges-of-up-scaling-lentivirus-production-and-processing
#5
REVIEW
Alexandra McCarron, Martin Donnelley, Chantelle McIntyre, David Parsons
Lentiviruses are becoming an increasingly popular choice of gene transfer vehicle for use in the treatment of a variety of genetic and acquired human diseases. As research progresses from basic studies into pre-clinical and clinical phases, there is a growing demand for large volumes of high purity, concentrated vector, and accordingly, the means to produce such quantities. Unlike other viral vectors, lentiviruses are difficult to produce using stable cell lines, therefore transient transfection of adherent cell lines is conventionally used, and this method has proven challenging to up-scale...
October 18, 2016: Journal of Biotechnology
https://www.readbyqxmd.com/read/27756280/lentivirus-mediated-short-hairpin-rna-interference-targeting-tnf-alpha-in-macrophages-inhibits-particle-induced-inflammation-and-osteolysis-in-vitro-and-in-vivo
#6
Chu-Qiang Qin, Dong-Sheng Huang, Chi Zhang, Bin Song, Jian-Bin Huang, Yue Ding
BACKGROUND: Aseptic loosening is a significant impediment to joint implant longevity. Prosthetic wear particles are postulated to play a central role in the onset and progression of periprosthetic osteolysis, leading to aseptic loosening of the prosthesis. METHODS: We investigated the inhibitory effects of a lentivirus-mediated short hairpin RNA that targets the TNF-alpha gene on the particle-induced inflammatory and osteolytic changes via macrophages both in vitro and in vivo...
October 18, 2016: BMC Musculoskeletal Disorders
https://www.readbyqxmd.com/read/27748828/effect-of-survivin-gene-therapy-via-lentivirus-vector-on-the-course-of-intervertebral-disc-degeneration-in-an-in%C3%A2-vivo-rabbit-model
#7
Bin Yue, Yazhou Lin, Xuexiao Ma, Guoqing Zhang, Bohua Chen
The aim of the current study was to use gene therapy to attenuate or reverse the degenerative process within the intervertabral disc. The effect of survivin gene therapy via lentiviral vector transfection on the course of intervertebral disc degeneration was investigated in the current study in an in vivo rabbit model. A total of 15 skeletally mature female New Zealand White rabbits were randomly divided into three groups: Punctured blank control group (group A, n=5), punctured empty vector control group (group B, n=5) and the treatment group (group C, n=5)...
November 2016: Molecular Medicine Reports
https://www.readbyqxmd.com/read/27742844/endothelial-cells-promote-expansion-of-long-term-engrafting-marrow-hematopoietic-stem-and-progenitor-cells-in-primates
#8
Jennifer L Gori, Jason M Butler, Balvir Kunar, Michael G Poulos, Michael Ginsberg, Daniel J Nolan, Zachary K Norgaard, Jennifer E Adair, Shahin Rafii, Hans-Peter Kiem
: : Successful expansion of bone marrow (BM) hematopoietic stem and progenitor cells (HSPCs) would benefit many HSPC transplantation and gene therapy/editing applications. However, current expansion technologies have been limited by a loss of multipotency and self-renewal properties ex vivo. We hypothesized that an ex vivo vascular niche would provide prohematopoietic signals to expand HSPCs while maintaining multipotency and self-renewal. To test this hypothesis, BM autologous CD34(+) cells were expanded in endothelial cell (EC) coculture and transplanted in nonhuman primates...
October 14, 2016: Stem Cells Translational Medicine
https://www.readbyqxmd.com/read/27729044/retroviral-vectors-and-transposons-for-stable-gene-therapy-advances-current-challenges-and-perspectives
#9
José Eduardo Vargas, Leonardo Chicaybam, Renato Tetelbom Stein, Amilcar Tanuri, Andrés Delgado-Cañedo, Martin H Bonamino
Gene therapy protocols require robust and long-term gene expression. For two decades, retrovirus family vectors have offered several attractive properties as stable gene-delivery vehicles. These vectors represent a technology with widespread use in basic biology and translational studies that require persistent gene expression for treatment of several monogenic diseases. Immunogenicity and insertional mutagenesis represent the main obstacles to a wider clinical use of these vectors. Efficient and safe non-viral vectors are emerging as a promising alternative and facilitate clinical gene therapy studies...
October 12, 2016: Journal of Translational Medicine
https://www.readbyqxmd.com/read/27667178/improvement-of-vascular-function-by-magnetic-nanoparticle-assisted-circumferential-gene-transfer-into-the-native-endothelium
#10
Sarah Vosen, Sarah Rieck, Alexandra Heidsieck, Olga Mykhaylyk, Katrin Zimmermann, Christian Plank, Bernhard Gleich, Alexander Pfeifer, Bernd K Fleischmann, Daniela Wenzel
Gene therapy is a promising approach for chronic disorders that require continuous treatment such as cardiovascular disease. Overexpression of vasoprotective genes has generated encouraging results in animal models, but not in clinical trials. One major problem in humans is the delivery of sufficient amounts of genetic vectors to the endothelium which is impeded by blood flow, whereas prolonged stop-flow conditions impose the risk of ischemia. In the current study we have therefore developed a strategy for the efficient circumferential lentiviral gene transfer in the native endothelium under constant flow conditions...
November 10, 2016: Journal of Controlled Release: Official Journal of the Controlled Release Society
https://www.readbyqxmd.com/read/27611239/lentivirus-mediated-correction-of-artemis-deficient-severe-combined-immunodeficiency
#11
Divya Punwani, Misako Kawahara, Jason Yu, Ukina Sanford, Sushmita Roy, Kiran Patel, Denise A Carbonaro, Andrea D Karlen, Sara Khan, Kenneth G Cornetta, Michael Rothe, Axel Schambach, Donald B Kohn, Harry L Malech, R Scott McIvor, Jennifer M Puck, Morton J Cowan
During B and T lymphocyte maturation, V(D)J recombination is initiated by creation of DNA double-strand breaks. Artemis is an exonuclease essential for their subsequent repair by non-homologous end-joining. Mutations in DCLRE1C, the gene encoding Artemis, cause T-B-NK+ severe combined immunodeficiency (ART-SCID) and also confer heightened sensitivity to ionizing radiation and alkylating chemotherapy. Although allogeneic hematopoietic cell transplantation (HCT) can treat ART-SCID, conditioning regimens are poorly tolerated, leading to early mortality and/or late complications, including short stature, endocrinopathies, and dental aplasia...
September 9, 2016: Human Gene Therapy
https://www.readbyqxmd.com/read/27573304/use-of-rhenium-188-for-in-vivo-imaging-and-treatment-of-human-cervical-cancer-cells-transfected-with-lentivirus-expressing-sodium-iodide-symporter
#12
Min Zhang, Shuo Shi, Rui Guo, Yin Miao, Biao Li
Although survival rates for cervical cancer have improved, they need further improvement in patients with distant metastases. The sodium iodine symporter (NIS) gene has often been used in cancer therapy and imaging. We examined the therapeutic effects of rhenium-188 (188Re) in a cervical cancer xenograft model expressing the NIS gene under the control of the tumor-specific human telomerase reverse transcriptase (hTERT) promoter. We constructed two recombinant lentiviral vectors expressing enhanced green fluorescent protein (eGFP) or the NIS gene driven by the hTERT promoter...
October 2016: Oncology Reports
https://www.readbyqxmd.com/read/27572655/promoter-optimisation-of-lentiviral-vectors-for-efficient-insulin-gene-expression-in-canine-mesenchymal-stromal-cells-potential-surrogate-beta-cells
#13
Pratigya Gautam, Robert D Foale, Asha Recino, Jing Zhao, Shu Uin Gan, Maja Wallberg, Roy Calne, Andrew M L Lever
BACKGROUND: The lack of an ideal cell type that can be easily acquired, modified to produce insulin, and re-implanted has been a limitation for ex vivo insulin gene therapy. Canine diabetes is currently treated with human insulin and is a good model for human diabetes. Mesenchymal stromal cells (MSCs) are a promising candidate cell type for gene therapy. Here we optimised insulin production using lentiviral transduced canine MSCs aiming to evaluate their ability for use as surrogate beta cells...
August 30, 2016: Journal of Gene Medicine
https://www.readbyqxmd.com/read/27519904/suppression-of-the-inflammatory-response-by-disease-inducible-interleukin-10-gene-therapy-in-a-three-dimensional-micromass-model-of-the-human-synovial-membrane
#14
Mathijs G A Broeren, Marieke de Vries, Miranda B Bennink, Onno J Arntz, Peter L E M van Lent, Peter M van der Kraan, Wim B van den Berg, Frank H J van den Hoogen, Marije I Koenders, Fons A J van de Loo
BACKGROUND: Gene therapy has the potential to provide long-term production of therapeutic proteins in the joints of osteoarthritis (OA) patients. The objective of this study was to analyse the therapeutic potential of disease-inducible expression of anti-inflammatory interleukin-10 (IL-10) in the three-dimensional micromass model of the human synovial membrane. METHODS: Synovial tissue samples from OA patients were digested and the cells were mixed with Matrigel to obtain 3D micromasses...
2016: Arthritis Research & Therapy
https://www.readbyqxmd.com/read/27490042/lentiviral-hsv-tk-007-mediated-suicide-gene-therapy-is-not-toxic-for-normal-brain-cells
#15
Jubayer A Hossain, Lars Rømo Ystaas, Jelena Mrdalj, Kristjan Välk, Kristoffer Riecken, Boris Fehse, Rolf Bjerkvig, Janne Grønli, Hrvoje Miletic
BACKGROUND: Gene therapeutic strategies with suicide genes are currently investigated in clinical trials for brain tumors. Previously, we have shown that lentiviral vectors delivering the suicide gene HSV-Tk to experimental brain tumors promote a highly significant treatment effect and thus are promising vectors for clinical translation. METHODS: In the present study, we tested lentiviral vectors delivering the suicide gene HSV-Tk.007, a highly active mutant of HSV-Tk, to rat brains as a preclinical toxicity study...
September 2016: Journal of Gene Medicine
https://www.readbyqxmd.com/read/27404517/stem-cell-based-engineered-immunity-against-hiv-infection-in-the-humanized-mouse-model
#16
Anjie Zhen, Valerie Rezek, Cindy Youn, Jonathan Rick, Brianna Lam, Nelson Chang, Jerome Zack, Masakazu Kamata, Scott Kitchen
With the rapid development of stem cell-based gene therapies against HIV, there is pressing requirement for an animal model to study the hematopoietic differentiation and immune function of the genetically modified cells. The humanized Bone-marrow/Liver/Thymus (BLT) mouse model allows for full reconstitution of a human immune system in the periphery, which includes T cells, B cells, NK cells and monocytes. The human thymic implant also allows for thymic selection of T cells in autologous thymic tissue. In addition to the study of HIV infection, the model stands as a powerful tool to study differentiation, development and functionality of cells derived from hematopoietic stem cells (HSCs)...
2016: Journal of Visualized Experiments: JoVE
https://www.readbyqxmd.com/read/27387837/assembly-pcr-synthesis-of-optimally-designed-compact-multi-responsive-promoters-suited-to-gene-therapy-application
#17
H Mohamed, Y Chernajovsky, D Gould
Gene therapy has the potential to provide innovative treatments for genetic and non-genetic diseases, with the ability to auto-regulate expression levels of therapeutic molecules so that they are produced locally and in direct response to disease activity. Generating disease responsive gene therapy vectors requires knowledge of the activation profile of transcription factors (TFs) during active disease, in order to assemble binding sites for these TFs into synthetic promoters, which can be appropriately activated by the disease process...
2016: Scientific Reports
https://www.readbyqxmd.com/read/27354226/transduction-of-hematopoietic-stem-cells-to-stimulate-rna-interference-against-feline-infectious-peritonitis
#18
Eman A Anis, Madhu Dhar, Alfred M Legendre, Rebecca P Wilkes
OBJECTIVES: The goals of the study were: (1) to develop and evaluate non-replicating lentivirus vectors coding for feline coronavirus (FCoV)-specific micro (mi)RNA as a potential antiviral therapy for feline infectious peritonitis (FIP); (2) to assess the feasibility of transducing hematopoietic stem cells (HSCs) with ex vivo introduction of the miRNA-expressing lentivirus vector; and (3) to assess the ability of the expressed miRNA to inhibit FCoV replication in HSCs in vitro. METHODS: HSCs were obtained from feline bone marrow and replicated in vitro...
June 27, 2016: Journal of Feline Medicine and Surgery
https://www.readbyqxmd.com/read/27333595/paralleled-comparison-of-vectors-for-the-generation-of-car-t-cells
#19
Di-Yuan Qin, Yong Huang, Dan Li, Yong-Sheng Wang, Wei Wang, Yu-Quan Wei
T-lymphocytes genetically engineered with the chimeric antigen receptor (CAR-T) have shown great therapeutic potential in cancer treatment. A variety of preclinical researches and clinical trials of CAR-T therapy have been carried out to lay the foundation for future clinical application. In these researches, several gene-transfer methods were used to deliver CARs or other genes into T-lymphocytes, equipping CAR-modified T cells with a property of recognizing and attacking antigen-expressing tumor cells in a major histocompatibility complex-independent manner...
September 2016: Anti-cancer Drugs
https://www.readbyqxmd.com/read/27317180/transient-expression-of-green-fluorescent-protein-in-integrase-defective-lentiviral-vector-transduced-293t-cell-line
#20
Fazlina Nordin, Zariyantey Abdul Hamid, Lucas Chan, Farzin Farzaneh, M K Azaham A Hamid
Non-integrating lentiviral vectors or also known as integrase-defective lentiviral (IDLV) hold a great promise for gene therapy application. They retain high transduction efficiency for efficient gene transfer in various cell types both in vitro and in vivo. IDLV is produced via a combined mutations introduced on the HIV-based lentiviral to disable their integration potency. Therefore, IDLV is considered safer than the wild-type integrase-proficient lentiviral vector as they could avoid the potential insertional mutagenesis associated with the nonspecific integration of transgene into target cell genome afforded by the wild-type vectors...
2016: Methods in Molecular Biology
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