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Molecular Therapy: the Journal of the American Society of Gene Therapy

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https://www.readbyqxmd.com/read/29339014/development-of-gpc3-specific-chimeric-antigen-receptor-engineered-natural-killer-cells-for-the-treatment-of-hepatocellular-carcinoma
#1
Min Yu, Hong Luo, Mingliang Fan, Xiuqi Wu, Bizhi Shi, Shengmeng Di, Ying Liu, Zeyan Pan, Hua Jiang, Zonghai Li
Chimeric antigen receptor (CAR)-modified natural killer (NK) cells represent a promising immunotherapeutic modality for cancer treatment. However, their potential utilities have not been explored in hepatocellular carcinoma (HCC). Glypian-3 (GPC3) is a rational immunotherapeutic target for HCC. In this study, we developed GPC3-specific NK cells and explored their potential in the treatment of HCC. The NK-92/9.28.z cell line was established by engineering NK-92, a highly cytotoxic NK cell line with second-generation GPC3-specific CAR...
December 19, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29273500/seek-and-you-will-not-find-ending-the-hunt-for-replication-competent-retroviruses-during-human-gene-therapy
#2
EDITORIAL
Helen E Heslop, Malcolm K Brenner
No abstract text is available yet for this article.
December 19, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29292164/gene-therapy-with-bmn-270-results-in-therapeutic-levels-of-fviii-in-mice-and-primates-and-normalization-of-bleeding-in-hemophilic-mice
#3
Stuart Bunting, Lening Zhang, Lin Xie, Sherry Bullens, Rajeev Mahimkar, Sylvia Fong, Krystal Sandza, Danielle Harmon, Bridget Yates, Britta Handyside, Choong-Ryoul Sihn, Nicole Galicia, Laurie Tsuruda, Charles A O'Neill, Anil Bagri, Peter Colosi, Shinong Long, Gordon Vehar, Barrie Carter
Hemophilia A is an X-linked bleeding disorder caused by mutations in the gene encoding the factor VIII (FVIII) coagulation protein. Bleeding episodes in patients are reduced by prophylactic therapy or treated acutely using recombinant or plasma-derived FVIII. We have made an adeno-associated virus 5 vector containing a B domain-deleted (BDD) FVIII gene (BMN 270) with a liver-specific promoter. BMN 270 injected into hemophilic mice resulted in a dose-dependent expression of BDD FVIII protein and a corresponding correction of bleeding time and blood loss...
December 14, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29249396/shed-skin-cancer-not-collagen-xvii-a-new-approach-to-targeting-skin-cancer-progression
#4
Olivier Gaide, Daniel Hohl
No abstract text is available yet for this article.
December 14, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29249394/vanadium-a-panacea-for-resistance-to-oncolytic-immunotherapy
#5
Dmitriy Zamarin
No abstract text is available yet for this article.
December 14, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29310916/chimeric-antigen-receptor-engineered-human-gamma-delta-t-cells-enhanced-cytotoxicity-with-retention-of-cross-presentation
#6
Anna Capsomidis, Gabriel Benthall, Heleen H Van Acker, Jonathan Fisher, Anne M Kramer, Zarah Abeln, Yvonne Majani, Talia Gileadi, Rebecca Wallace, Kenth Gustafsson, Barry Flutter, John Anderson
Gamma delta T (γδT) lymphocytes are primed for rapid function, including cytotoxicity toward cancer cells, and are a component of the immediate stress response. Following activation, they can function as professional antigen-presenting cells. Chimeric antigen receptors (CARs) work by focusing T cell function on defined cell surface tumor antigens and provide essential costimulation for robust activation. Given the natural tropism of γδT cells for the tumor microenvironment, we hypothesized that their transduction with CARs might enhance cytotoxicity while retaining their ability to migrate to tumor and act as antigen-presenting cells to prolong the intratumoral immune response...
December 8, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29292162/aav8-antivegffab-ocular-gene-transfer-for-neovascular-age-related-macular-degeneration
#7
Yuanyuan Liu, Seth D Fortmann, Jikui Shen, Erik Wielechowski, Anna Tretiakova, Stephen Yoo, Karen Kozarsky, Jiangxia Wang, James M Wilson, Peter A Campochiaro
Sustained suppression of VEGF is needed in many patients with neovascular age-related macular degeneration (NVAMD), and gene transfer of a VEGF-neutralizing protein is a promising approach to achieve it. Initial clinical trials testing this approach have shown encouraging signals, but evidence of robust transgene expression and consistent antiangiogenic and antipermeability activity has been lacking. In this study, we demonstrate expression of an anti-human VEGF antibody fragment (antiVEGFfab) after subretinal injection of AAV8-antiVEGFfab...
December 8, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29331291/global-microrna-profiling-in-human-bone-marrow-skeletal-stromal-or-mesenchymal-stem-cells-identified-candidates-for-bone-regeneration
#8
Chi-Chih Chang, Morten T Venø, Li Chen, Nicholas Ditzel, Dang Q S Le, Philipp Dillschneider, Moustapha Kassem, Jørgen Kjems
Bone remodeling and regeneration are highly regulated multistep processes involving posttranscriptional regulation by microRNAs (miRNAs). Here, we performed a global profiling of differentially expressed miRNAs in bone-marrow-derived skeletal cells (BMSCs; also known as stromal or mesenchymal stem cells) during in vitro osteoblast differentiation. We functionally validated the regulatory effects of several miRNAs on osteoblast differentiation and identified 15 miRNAs, most significantly miR-222 and miR-423, as regulators of osteoblastogenesis...
December 5, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29292161/triple-vectors-expand-aav-transfer-capacity-in-the-retina
#9
Andrea Maddalena, Patrizia Tornabene, Paola Tiberi, Renato Minopoli, Anna Manfredi, Margherita Mutarelli, Settimio Rossi, Francesca Simonelli, Jurgen K Naggert, Davide Cacchiarelli, Alberto Auricchio
Retinal gene transfer with adeno-associated viral (AAV) vectors holds great promise for the treatment of inherited retinal degenerations (IRDs). One limit of AAV is its transfer capacity of about 5 kb, which can be expanded to about 9 kb, using dual AAV vectors. This strategy would still not suffice for treatment of IRDs such as Usher syndrome type 1D or Alström syndrome type I (ALMS) due to mutations in CDH23 or ALMS1, respectively. To overcome this limitation, we generated triple AAV vectors, with a maximal transfer capacity of about 14 kb...
December 5, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29275847/self-amplifying-rna-vaccines-give-equivalent-protection-against-influenza-to-mrna-vaccines-but-at-much-lower-doses
#10
Annette B Vogel, Laura Lambert, Ekaterina Kinnear, David Busse, Stephanie Erbar, Kerstin C Reuter, Lena Wicke, Mario Perkovic, Tim Beissert, Heinrich Haas, Stephen T Reece, Ugur Sahin, John S Tregoning
New vaccine platforms are needed to address the time gap between pathogen emergence and vaccine licensure. RNA-based vaccines are an attractive candidate for this role: they are safe, are produced cell free, and can be rapidly generated in response to pathogen emergence. Two RNA vaccine platforms are available: synthetic mRNA molecules encoding only the antigen of interest and self-amplifying RNA (sa-RNA). sa-RNA is virally derived and encodes both the antigen of interest and proteins enabling RNA vaccine replication...
December 5, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29258739/combination-immunotherapy-of-muc1-mrna-nano-vaccine-and-ctla-4-blockade-effectively-inhibits-growth-of-triple-negative-breast-cancer
#11
Lina Liu, Yuhua Wang, Lei Miao, Qi Liu, Sara Musetti, Jun Li, Leaf Huang
Triple negative breast cancer (TNBC), which constitutes 10%-20% of all breast cancers, is associated with aggressive progression, a high rate of metastasis, and poor prognosis. The treatment of patients with TNBC remains a great clinical challenge. Preclinical reports support the combination immunotherapy of cancer vaccines and immune checkpoint blockades in non-immunogenic tumors. In this study, we constructed nanoparticles (NPs) to deliver an mRNA vaccine encoding tumor antigen MUC1 to dendritic cells (DCs) in lymph nodes to activate and expand tumor-specific T cells...
December 5, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29292163/hypoxic-lung-cancer-derived-extracellular-vesicle-microrna-103a-increases-the-oncogenic-effects-of-macrophages-by-targeting-pten
#12
Ya-Ling Hsu, Jen-Yu Hung, Wei-An Chang, Shu-Fang Jian, Yi-Shiuan Lin, Yi-Chung Pan, Cheng-Ying Wu, Po-Lin Kuo
Hypoxia, the most commonly observed characteristic in cancers, is implicated in the establishment of an immunosuppressive niche. Recent studies have indicated that extracellular vesicle (EV)-mediated cancer-stroma interactions are considered to play a critical role in the regulation of various cellular biological functions, with phenotypic consequences in recipient cells. However, the mechanisms underlying the relationship between EVs and hypoxia during cancer progression remain largely unknown. In this study, we found that EVs derived from hypoxic lung cancers increased M2-type polarization by miR-103a transfer...
November 29, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29273501/selective-%C3%AE-synuclein-knockdown-in-monoamine-neurons-by-intranasal-oligonucleotide-delivery-potential-therapy-for-parkinson-s-disease
#13
Diana Alarcón-Arís, Ariadna Recasens, Mireia Galofré, Iria Carballo-Carbajal, Nicolás Zacchi, Esther Ruiz-Bronchal, Rubén Pavia-Collado, Rosario Chica, Albert Ferrés-Coy, Marina Santos, Raquel Revilla, Andrés Montefeltro, Isabel Fariñas, Francesc Artigas, Miquel Vila, Analia Bortolozzi
Progressive neuronal death in brainstem nuclei and widespread accumulation of α-synuclein are neuropathological hallmarks of Parkinson's disease (PD). Reduction of α-synuclein levels is therefore a potential therapy for PD. However, because α-synuclein is essential for neuronal development and function, α-synuclein elimination would dramatically impact brain function. We previously developed conjugated small interfering RNA (siRNA) sequences that selectively target serotonin (5-HT) or norepinephrine (NE) neurons after intranasal administration...
November 29, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29196135/esgct-2017-in-berlin-clear-steps-forward
#14
EDITORIAL
Seppo Ylä-Herttuala
No abstract text is available yet for this article.
November 28, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29275848/mir-23b-suppresses-leukocyte-migration-and-pathogenesis-of-experimental-autoimmune-encephalomyelitis-by-targeting-ccl7
#15
Yuan Zhang, Juan-Juan Han, Xiao-Yan Liang, Li Zhao, Fei Zhang, Javad Rasouli, Zhe-Zhi Wang, Guang-Xian Zhang, Xing Li
MicroRNAs (miRNAs) are small, non-coding RNAs involved in immune response regulation. Specific miRNAs have been linked to the development of various autoimmune diseases; however, their contribution to the modulation of CNS-directed cellular infiltration remains unclear. In this study, we found that miR-23b, in addition to its reported functions in the suppression of IL-17-associated autoimmune inflammation, halted the progression of experimental autoimmune encephalomyelitis (EAE), an animal model of multiple sclerosis (MS), by directly inhibiting the migration of pathogenic leukocytes to the CNS...
November 26, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29273502/mir-25-tough-decoy-enhances-cardiac-function-in-heart-failure
#16
Dongtak Jeong, Jimeen Yoo, Philyoung Lee, Sacha V Kepreotis, Ahyoung Lee, Christine Wahlquist, Brian D Brown, Changwon Kho, Mark Mercola, Roger J Hajjar
MicroRNAs are promising therapeutic targets, because their inhibition has the potential to normalize gene expression in diseased states. Recently, our group found that miR-25 is a key SERCA2a regulating microRNA, and we showed that multiple injections of antagomirs against miR-25 enhance cardiac contractility and function through SERCA2a restoration in a murine heart failure model. However, for clinical application, a more stable suppressor of miR-25 would be desirable. Tough Decoy (TuD) inhibitors are emerging as a highly effective method for microRNA inhibition due to their resistance to endonucleolytic degradation, high miRNA binding affinity, and efficient delivery...
November 26, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29273498/engineering-protein-secreting-plasma-cells-by-homology-directed-repair-in-primary-human-b-cells
#17
King L Hung, Iana Meitlis, Malika Hale, Chun-Yu Chen, Swati Singh, Shaun W Jackson, Carol H Miao, Iram F Khan, David J Rawlings, Richard G James
The ability to engineer primary human B cells to differentiate into long-lived plasma cells and secrete a de novo protein may allow the creation of novel plasma cell therapies for protein deficiency diseases and other clinical applications. We initially developed methods for efficient genome editing of primary B cells isolated from peripheral blood. By delivering CRISPR/CRISPR-associated protein 9 (Cas9) ribonucleoprotein (RNP) complexes under conditions of rapid B cell expansion, we achieved site-specific gene disruption at multiple loci in primary human B cells (with editing rates of up to 94%)...
November 22, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29174842/oligonucleotide-therapies-for-the-lung-ready-to-return-to-the-clinic
#18
Manish Kumar, Sterghios A Moschos
No abstract text is available yet for this article.
November 22, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29249397/mrna-vaccine-with-antigen-specific-checkpoint-blockade-induces-an-enhanced-immune-response-against-established-melanoma
#19
Yuhua Wang, Lu Zhang, Zhenghong Xu, Lei Miao, Leaf Huang
We reported a preclinical cancer vaccine that simultaneously introduced an mRNA antigen and an immune checkpoint blocking siRNA into the antigen-presenting cells. This was achieved by formulating both nucleic acid-based immunotherapeutics into a lipid-coated calcium phosphate (LCP) nanoparticle (NP) as a carrier to address the delivery challenge. The PEGylated lipid NPs were functionalized with mannose as the targeting ligand to facilitate the preferential uptake by the dendritic cells (DCs) in the lymph nodes after subcutaneous administration...
November 21, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29249395/synergy-of-immune-checkpoint-blockade-with-a-novel-synthetic-consensus-dna-vaccine-targeting-tert
#20
Elizabeth K Duperret, Megan C Wise, Aspen Trautz, Daniel O Villarreal, Bernadette Ferraro, Jewell Walters, Jian Yan, Amir Khan, Emma Masteller, Laurent Humeau, David B Weiner
Immune checkpoint blockade antibodies are setting a new standard of care for cancer patients. It is therefore important to assess any new immune-based therapies in the context of immune checkpoint blockade. Here, we evaluate the impact of combining a synthetic consensus TERT DNA vaccine that has improved capacity to break tolerance with immune checkpoint inhibitors. We observed that blockade of CTLA-4 or, to a lesser extent, PD-1 synergized with TERT vaccine, generating more robust anti-tumor activity compared to checkpoint alone or vaccine alone...
November 21, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
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