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Human Gene Therapy

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https://www.readbyqxmd.com/read/29037080/esgct-xxv-anniversary-congress-in-collaboration-with-the-german-society-for-gene-therapy-october-17-20-2017-berlin-germany
#1
(no author information available yet)
No abstract text is available yet for this article.
October 16, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/29037077/esgct-xxv-anniversary-congress-in-collaboration-with-the-german-society-for-gene-therapy-abstract-author-index
#2
(no author information available yet)
No abstract text is available yet for this article.
October 16, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28990426/gene-therapy-for-modulation-of-t-cell-mediated-immune-response-provoked-by-corneal-transplantation
#3
Marko Pastak, Veronika Kleff, Daniel Raphael Saban, Marta Czugala, Klaus-Peter Steuhl, Süleyman Ergün, Bernhard Singer, Thomas Armin Fuchsluger
Corneal transplantation (=keratoplasty) is the most common type of tissue replacement in the world. The increased rate of graft rejection after keratoplasty is a central problem for repeated transplantations and in inflamed host corneas. It has been shown that apoptosis of grafted epithelium has a role in corneal allograft rejection. Our study focused on the T cell response triggered in BALB/c mice after allogeneic corneal transplantation with and without anti-apoptotic p35-transduced epithelium. To restrict p35 expression to the epithelial cells we created modified allogeneic composite grafts...
October 7, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28990424/an-inducible-promoter-responsive-to-different-porphyrinogenic-stimuli-improves-gene-therapy-vectors-for-acute-intermittent-porphyria
#4
Irantzu Serrano, Ana Sampedro, Manuel Alegre, Rafael Enriquez de Salamanca, Pedro Berraondo, Antonio Fontanellas
Porphobilinogen deaminase (PBGD)-gene therapy represents a promising therapeutic option for acute intermittent porphyria (AIP) patients suffering recurrent acute attacks. The first-in-human phase 1 clinical trial confirmed the safety and tolerability of AAV-AAT-PBGD gene therapy but higher doses and/or more efficient vectors are needed to achieve therapeutic expression of the transgene. We assayed the insertion into the promoter of a short enhancer element able to induce transgene expression during the exposure to endogenous and exogenous stimuli related to the pathology of the disease...
October 7, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28806897/evaluation-of-intrathecal-routes-of-administration-for-adeno-associated-viral-vectors-in-large-animals
#5
Christian Hinderer, Peter Bell, Nathan Katz, Charles H Vite, Jean-Pierre Louboutin, Erin Bote, Hongwei Yu, Yanqing Zhu, Margret L Casal, Jessica Bagel, Patricia O'Donnell, Ping Wang, Mark E Haskins, Tamara Goode, James M Wilson
Delivery of adeno-associated viral (AAV) vectors into the cerebrospinal fluid (CSF) can achieve gene transfer to cells throughout the brain and spinal cord, potentially making many neurological diseases tractable gene therapy targets. Identifying the optimal route of CSF access for intrathecal AAV delivery will be a critical step in translating this approach to clinical practice. We previously demonstrated that vector injection into the cisterna magna is a safe and effective method for intrathecal AAV delivery in nonhuman primates; however, this procedure is not commonly used in clinical practice...
October 3, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28726522/steerable-induction-of-the-thymosin-%C3%AE-4-mrtf-a-pathway-via-aav-based-overexpression-induces-therapeutic-neovascularization
#6
Tilman Ziegler, Markus Kraus, Wira Husada, Florian Gesenhues, Qui Jiang, Olaf Pinkenburg, Teresa Trenkwalder, Karl-Ludwig Laugwitz, Ferdinand le Noble, Christian Weber, Christian Kupatt, Rabea Hinkel
Viral vectors have been frequently used in a variety of preclinical animal models to deliver genetic constructs into tissues. Among the vectors used, adeno-associated viral vectors (AAVs) may be targeted to specific tissues, depending on the serotype used. Moreover, they show robust expression for prolonged periods of time and have a low immunogenic potential. Furthermore, AAVs, unlike other vector systems, only display a low rate of genomic integration. However, to ensure efficient transgene production, expression is typically driven by constitutively active promoters, such as the cytomegalovirus (CMV) promoter...
October 3, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28726516/targeting-nsg-mice-engrafting-cells-with-a-clinically-applicable-lentiviral-vector-corrects-osteoclasts-in-infantile-malignant-osteopetrosis
#7
Ilana Moscatelli, Henrik Löfvall, Christian Schneider Thudium, Michael Rothe, Carmen Montano, Zsuzsanna Kertész, Mehtap Sirin, Ansgar Schulz, Axel Schambach, Kim Henriksen, Johan Richter
Infantile malignant osteopetrosis (IMO) is a rare, lethal, autosomal recessive disorder characterized by nonfunctional osteoclasts. More than 50% of the patients have mutations in the TCIRG1 gene, encoding for a subunit of the osteoclast proton pump. The aim of this study was to develop a clinically applicable lentiviral vector expressing TCIRG1 to correct osteoclast function in IMO. Two mammalian promoters were compared: elongation factor 1α short (EFS) promoter and chimeric myeloid promoter (ChimP). EFS promoter was chosen for continued experiments, as it performed better...
October 3, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28967304/systemic-smad7-gene-therapy-increases-striated-muscle-mass-and-enhances-exercise-capacity-in-a-dose-dependent-manner
#8
Joseph Maricelli, Yemeserach Bishaw, Bo Wang, Min Du, Buel Dantese Rodgers
Striated muscle wasting occurs with a variety of disease indications, contributing to mortality and compromising life quality. Recent studies indicate that the recombinant adeno-associated viral (serotype 6) Smad7 gene therapeutic, AVGN7, enhances skeletal and cardiac muscle mass and prevents cancer-induced wasting of both tissues. This is accomplished by attenuating ActRIIb intracellular signaling and as a result, the physiological actions of myostatin and other ActRIIb ligands. AVGN7 also enhances isolated skeletal muscle twitch force, but is unknown to similarly improve systemic muscle function, especially exercise capacity...
October 1, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28950731/unbiased-identification-of-t-cell-receptors-targeting-immunodominant-peptide-mhc-complexes-for-t-cell-receptor-immunotherapy
#9
Felix K M Lorenz, Christian Ellinger, Elisa Kieback, Susanne Wilde, Maria Lietz, Dolores J Schendel, Wolfgang Uckert
T cell receptor (TCR) immunotherapy uses T cells engineered with new TCRs to enable detection and killing of cancer cells. Efficacy of TCR immunotherapy depends on targeting antigenic peptides that are efficiently presented by the best suited major histocompatibility complex (MHC) molecules of cancer cells. However, efficient strategies are lacking to easily identify TCRs recognizing immunodominant peptide-MHC (pMHC) combinations utilizing any of the six possible MHC class I alleles of a cancer cell. We generated an MHC cell library and developed a platform approach to detect, isolate and re-express TCRs specific for immunodominant pMHCs...
September 26, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28950723/transplantation-of-hgf-modified-dental-pulp-stem-cells-prevents-bone-loss-in-the-early-phase-of-ovariectomy-induced-osteoporosis
#10
Fanxuan Kong, Xuefeng Shi, FengJun Xiao, Yuefeng Yang, Xiaoyan Zhang, Li-Sheng Wang, Chu-Tse Wu, Hua Wang
Investigations based on mesenchymal stem cells (MSCs) for osteoporosis have been obtained intensive attention recently. MSCs can derive from various tissues, such as bone marrow, adipose, umbilical cord, placenta, dental pulp, etc. Among them, dental pulp derived MSCs (DPSCs) and hepatocyte growth factor (HGF)-modified DPSCs (DPSCs-HGF) highly expressed osteogenic related genes and have stronger osteogenic differentiation capacities. DPSCs have more benefits in treating osteoporosis. The purpose of this study is to investigate the roles of HGF gene modified DPSCs in bone regeneration using a mouse model of ovariectomy (OVX)-induced bone loss...
September 26, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28922955/comparison-of-zfns-versus-crispr-specific-nucleases-for-genome-edition-of-the-wiskott-aldrich-syndrome-locus
#11
Alejandra Gutierrez-Guerrero, Sabina Sanchez-Hernandez, Giuseppe Galvani, Javier Pinedo-Gomez, Almudena Sanchez-Gilabert, Rocio Martin-Guerra, Marien Cobo, Philip Gregory, Michael Holmes, Karim Benabdellah, Francisco Martin
Primary immunodeficiencies (PID), including Wiskott-Aldrich syndrome (WAS), are a main target for genome editing (GE) strategies using specific nucleases (SNs) since a small number of corrected hematopoietic stem cells (HSCs) could cure patients. In this work, we have designed different WAS gene-specific CRISPR/Cas9 systems and compared their efficiency and specificity with homodimeric and heterodimeric WAS-specific Zinc Finger Nucleases (ZFNs) using K562 cells as a cellular model and plasmid nucleofection or integrative-deficient Lentiviral Vectors (IDLVs) for delivery...
September 19, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28922951/a-retrospective-study-of-cytokine-profiles-changes-in-mice-with-fviii-inhibitor-development-after-aav-mediated-gene-therapy-in-hemophilia-a-mouse-model
#12
Junjiang Sun, Zhenhua Yuan, Yasmina L Abajas, Dorreen E Szollosi, Genlin Hu, Baolai Hua, Xiao Xiao, Chengwen Li
The development of inhibitory autoantibodies to the infused clotting factor VIII is a major complication for severe hemophilia A management. Novel therapy options for hemophilia have significantly progressed in the last decade and a gene therapy cure for hemophilia is translating into reality. However, mechanistic studies of FVIII autoantibodies (FVIII inhibitors) have lagged behind and remain a challenge for both protein replacement and gene therapy. FVIII inhibitor formation is assumed to be a classical T cell-dependent immune response in which cytokines/chemokines play an important role...
September 19, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28922945/aav-gene-therapy-in-a-sheep-model-of-tay-sachs-disease
#13
Heather Gray-Edwards, Ashley N Randle, Stacy Maitland, Hector Benatti, Spencer Hubbard, Peter Canning, Matthew Vogel, Brandon Brunson, Misako Hwang, Lauren Ellis, Allison M Bradbury, Atoska Gentry, Amanda Taylor, Anne Wooldridge, Dewey Wilhite, Randoplh Winter, Brain Whitlock, Jacob A Johnson, Merrilee Holland, Nouha Salibi, Ronald Beyers, James Sartin, Thomas Denney, Nancy R Cox, Miguel Sena-Esteves, Douglas R Martin
Tay-Sachs disease (TSD) is a fatal neurodegenerative disorder caused by a deficiency of the enzyme hexosaminidase A (HexA). Tay-Sachs disease also occurs in sheep, the only experimental model of TSD that has clinical signs of disease. The natural history of sheep TSD was characterized using serial neurological evaluations, 7 tesla MRI, echocardiograms, electrodiagnostics and cerebrospinal fluid (CSF) biomarkers. Intracranial gene therapy was also tested using AAVrh8 monocistronic vectors encoding the α subunit of Hex (TSD α) or a mixture of two vectors encoding both the α and β subunits separately (TSD α+β) injected at high (1...
September 19, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28877647/improvement-of-in-vivo-expression-of-genes-delivered-by-self-amplifying-rna-using-vaccinia-virus-immune-evasion-proteins
#14
Tim Beissert, Lars Koste, Mario Perkovic, Kerstin C Walzer, Stephanie Erbar, Abderaouf Selmi, Mustafa Diken, Sebastian Kreiter, Özlem Türeci, Ugur Sahin
Among nucleic acid based delivery platforms, self-amplifying RNA (saRNA) vectors are of increasing interest for applications such as transient expression of recombinant proteins and vaccination. saRNA is safe and, due to its capability to amplify intracellularly high protein levels, can by produced from even minute amounts of transfected templates. It is an obstacle to full exploitation of this platform, though, that saRNA induces a strong innate host immune response. In transfected cells pattern recognition receptors sense double-stranded RNA intermediates and via activation of protein kinase R (PKR) and interferon signaling initiate host defense measures including a translational shut-down...
September 6, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28874076/multimodal-fluorescence-and-bioluminescence-imaging-reveals-transfection-potential-of-intratracheally-administered-polyplexes-for-breast-cancer-lung-metastases
#15
Antonia Geyer, Alexander Taschauer, Fatih Alioglu, Martina Anton, Julia Maier, Elisabeth Drothler, Manuela Simlinger, Sümeyye Yavuz, Haider Sami, Manfred Ogris
Local delivery of anticancer agents or gene therapeutics to lung tumors can circumvent side effects or accumulation in non-target organs, but accessibility via the alveolar side of blood-air barrier remains challenging. Polyplexes based on plasmid and linear polyethylenimine (LPEI) transfect healthy lung tissue when applied intravenously in the mouse, but direct delivery into the lungs results in low transfection of lung tissue. Nevertheless, LPEI could offer the potential to selectively transfect lung tumors, if accessible by the alveolar side...
September 6, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28870121/wide-awake-and-ready-to-move-20-years-of-non-viral-therapeutic-genome-engineering-with-the-sleeping-beauty-transposon-system
#16
Russ Hodge, Suneel Narayanavari, Zsuzsanna Izsvák, Zoltán Ivics
Gene therapies will only become a widespread tool in the clinical treatment of human diseases with the advent of gene transfer vectors that integrate genetic information stably, safely, effectively, and economically. Two decades after the discovery of the Sleeping Beauty (SB) transposon, it has been transformed into a vector system that is fulfilling these requirements. SB may well overcome some of the limitations associated with viral gene transfer vectors and transient non-viral gene delivery approaches that are being used in the majority of ongoing clinical trials...
September 4, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28870120/virotherapy-research-in-germany-from-engineering-to-translation-a-review-as-contribution-to-the-combined-annual-meeting-of-the-german-and-european-societies-of-gene-and-cell-therapy-2017
#17
Guy Ungerechts, Christine E Engeland, Christian J Buchholz, Jürgen Eberle, Henry Fechner, Karsten Geletneky, Per Sonne Holm, Florian Kreppel, Florian Kühnel, Karl Sebastian Lang, Mathias F Leber, Antonio Marchini, Markus Moehler, Michael D Mühlebach, Jean Rommelaere, Christoph Springfeld, Ulrich M Lauer, Dirk M Nettelbeck
Virotherapy is a unique modality for treatment of cancer with oncolytic viruses (OVs) that selectively infect and lyse tumor cells, spread within tumors, and activate anti-tumor immunity. Different viruses are being developed as OVs preclinically and clinically, several of them engineered to encode therapeutic proteins for tumor-targeted gene therapy. Scientists and clinicians in Germany have made significant contributions to OV research and development, which are highlighted in this review article. Innovative strategies for "shielding", entry- or post-entry targeting, and "arming" of OVs have been established focusing on adeno-, measles, parvo-, and vaccinia virus platforms...
September 4, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28859532/the-european-society-for-gene-and-cell-therapy-esgct-inaugural-learned-society-in-the-field-worldwide-a-vision-on-its-birth-life-and-prospects-for-sustainability
#18
https://www.readbyqxmd.com/read/28854824/promises-and-challenges-in-hematopoietic-stem-cell-gene-therapy
#19
Saskia Kohlscheen, Halvard Boenig, Ute Modlich
Hematopoietic stem cell-directed gene therapy (HSC-GT) provides an innovative treatment option for hematological disorders. Gene therapy promises to cure the disease "at the root" and is therefore exceptional in its potential, but also formidable in its challenges as long-term side effects are hard to predict and clinical experience remains limited. Many excellent reviews on the topic by designated experts in the field of HSC-GT have come forth, elucidating to the successes and pitfalls in the various clinical studies 1-3...
August 30, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28854818/recent-advances-in-preclinical-developments-using-adenovirus-hybrid-vectors
#20
Eric Ehrke-Schulz, Wenli Zhang, Jian Gao, Anja Ehrhardt
Adenovirus (Ad) based vectors are efficient gene transfer vehicles to deliver foreign DNA into living organisms offering large cargo capacity low immunogenicity and genotoxicity. As Ad shows low integration rates of their genomes into host chromosomes, vector derived gene expression decreases due to continuous cell cycling in regenerating tissues and dividing cell populations. To overcome this hurdle adenoviral delivery can be combined with mechanisms leading to maintenance of therapeutic DNA and long-term effects of the desired treatment...
August 30, 2017: Human Gene Therapy
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