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

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https://www.readbyqxmd.com/read/29784587/predicting-dangerous-rides-in-car-t-cells-bridging-the-gap-between-mice-and-humans
#1
Marco Ruella, Carl H June
No abstract text is available yet for this article.
May 18, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29778522/potential-therapeutic-impact-of-mir-145-deregulation-in-colorectal-cancer
#2
LETTER
Ion Cristóbal, Marta Sanz-Alvarez, Blanca Torrejón, Andrea Santos, Melani Luque, Federico Rojo, Jesús García-Foncillas
No abstract text is available yet for this article.
May 16, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29784585/inhibition-of-glycogen-synthase-ii-with-rnai-prevents-liver-injury-in-mouse-models-of-glycogen-storage-diseases
#3
Natalie Pursell, Jessica Gierut, Wei Zhou, Michael Dills, Rohan Diwanji, Monika Gjorgjieva, Utsav Saxena, Jr-Shiuan Yang, Anee Shah, Nandini Venkat, Rachel Storr, Boyoung Kim, Weimin Wang, Marc Abrams, Margaux Raffin, Gilles Mithieux, Fabienne Rajas, Henryk Dudek, Bob D Brown, Chengjung Lai
Glycogen storage diseases (GSDs) of the liver are devastating disorders presenting with fasting hypoglycemia as well as hepatic glycogen and lipid accumulation, which could lead to long-term liver damage. Diet control is frequently utilized to manage the potentially dangerous hypoglycemia, but there is currently no effective pharmacological treatment for preventing hepatomegaly and concurrent liver metabolic abnormalities, which could lead to fibrosis, cirrhosis, and hepatocellular adenoma or carcinoma. In this study, we demonstrate that inhibition of glycogen synthesis using an RNAi approach to silence hepatic Gys2 expression effectively prevents glycogen synthesis, glycogen accumulation, hepatomegaly, fibrosis, and nodule development in a mouse model of GSD III...
April 27, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29778523/local-immunomodulation-with-anti-inflammatory-cytokine-encoding-lentivirus-enhances-functional-recovery-after-spinal-cord-injury
#4
Jonghyuck Park, Joseph T Decker, Daniel J Margul, Dominique R Smith, Brian J Cummings, Aileen J Anderson, Lonnie D Shea
Trauma to the spinal cord and associated secondary inflammation can lead to permanent loss of sensory and motor function below the injury level, with the resulting environment serving as a barrier that limits regeneration. In this study, we investigate the localized expression of anti-inflammatory cytokines IL-10 and IL-4 via lentiviral transduction in multichannel bridges. Porous multichannel bridges provide physical guidance for axonal outgrowth with the cytokines hypothesized to modulate the neuroinflammatory microenvironment and enhance axonal regeneration...
April 27, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29784586/ultrasound-mediated-gene-delivery-enhances-tendon-allograft-integration-in-mini-pig-ligament-reconstruction
#5
Maxim Bez, Thomas J Kremen, Wafa Tawackoli, Pablo Avalos, Dmitriy Sheyn, Galina Shapiro, Joseph C Giaconi, Shiran Ben David, Jess G Snedeker, Zulma Gazit, Katherine W Ferrara, Dan Gazit, Gadi Pelled
Ligament injuries occur frequently, substantially hindering routine daily activities and sports participation in patients. Surgical reconstruction using autogenous or allogeneic tissues is the gold standard treatment for ligament injuries. Although surgeons routinely perform ligament reconstructions, the integrity of these reconstructions largely depends on adequate biological healing of the interface between the ligament graft and the bone. We hypothesized that localized ultrasound-mediated, microbubble-enhanced therapeutic gene delivery to endogenous stem cells would lead to significantly improved ligament graft integration...
April 26, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29759938/differentiation-therapy-by-epigenetic-reconditioning-exerts-antitumor-effects-on-liver-cancer-cells
#6
Luc Gailhouste, Lee Chuen Liew, Ken Yasukawa, Izuho Hatada, Yasuhito Tanaka, Hitoshi Nakagama, Takahiro Ochiya
Primary liver tumors are mainly represented by hepatocellular carcinoma (HCC), one of the most aggressive and resistant forms of cancer. Liver tumorigenesis is characterized by an accumulation of epigenetic abnormalities, leading to gene extinction and loss of hepatocyte differentiation. The aim of this work was to investigate the feasibility of converting liver cancer cells toward a less aggressive and differentiated phenotype using a process called epigenetic reconditioning. Here, we showed that an epigenetic regimen with non-cytotoxic doses of the demethylating compound 5-azacytidine (5-AZA) promoted an anti-cancer response by inhibiting HCC cell tumorigenicity...
April 26, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29759937/identification-of-epigenetic-regulators-of-dux4-fl-for-targeted-therapy-of-facioscapulohumeral-muscular-dystrophy
#7
Charis L Himeda, Takako I Jones, Ching-Man Virbasius, Lihua Julie Zhu, Michael R Green, Peter L Jones
Facioscapulohumeral muscular dystrophy (FSHD) is caused by epigenetic de-repression of the disease locus, leading to pathogenic misexpression of the DUX4 gene in skeletal muscle. While the factors and pathways involved in normal repression of the FSHD locus in healthy cells have been well characterized, very little is known about those responsible for the aberrant activation of DUX4-fl in FSHD myocytes. Reasoning that DUX4-fl activators might represent useful targets for small molecule inhibition, we performed a highly targeted, candidate-based screen of epigenetic regulators in primary FSHD myocytes...
April 26, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29778524/posttranscriptional-regulation-of-interleukin-33-expression-by-microrna-200-in-bronchial-asthma
#8
Xin Tang, Feng Wu, Jinshuo Fan, Yang Jin, Jianjun Wang, Guanghai Yang
The importance of understanding how interleukin-33 (IL-33) is regulated (particularly by miRs) is critical in IL-33 biology, and evidence of this in asthma pathology is limited. MicroRNA profiling of cells isolated from bronchoalveolar lavage of 14 asthmatic patients and 11 healthy controls revealed miR-200b and miR-200c were significantly reduced in asthmatic patients compared with healthy controls. The reduction was validated in two independent models of allergen-induced allergic airway inflammation and further demonstrated to be inversely correlated with asthma severity, as well as increased IL-33 production in asthmatic patients...
April 25, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29754775/in-situ-gene-therapy-via-aav-crispr-cas9-mediated-targeted-gene-regulation
#9
Ana M Moreno, Xin Fu, Jie Zhu, Dhruva Katrekar, Yu-Ru V Shih, John Marlett, Jessica Cabotaje, Jasmine Tat, John Naughton, Leszek Lisowski, Shyni Varghese, Kang Zhang, Prashant Mali
Development of efficacious in vivo delivery platforms for CRISPR-Cas9-based epigenome engineering will be critical to enable the ability to target human diseases without permanent modification of the genome. Toward this, we utilized split-Cas9 systems to develop a modular adeno-associated viral (AAV) vector platform for CRISPR-Cas9 delivery to enable the full spectrum of targeted in situ gene regulation functionalities, demonstrating robust transcriptional repression (up to 80%) and activation (up to 6-fold) of target genes in cell culture and mice...
April 25, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29685384/registry-contributions-to-strengthen-cell-and-gene-therapeutic-evidence
#10
Mohamed Abou-El-Enein, David W Grainger, Sven Kili
No abstract text is available yet for this article.
April 20, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29680696/aptamer-based-control-of-gene-expression-utilizing-endogenous-mirnas
#11
Jörg S Hartig
No abstract text is available yet for this article.
April 18, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29678655/not-all-reprogrammed-insulin-producing-cells-are-the-same
#12
Mingming Gao, Dexi Liu
No abstract text is available yet for this article.
April 17, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29730197/heat-shock-factor-1-epigenetically-stimulates-glutaminase-1-dependent-mtor-activation-to-promote-colorectal-carcinogenesis
#13
Jiaqiu Li, Ping Song, Tingting Jiang, Dongjun Dai, Hanying Wang, Jie Sun, Liyuan Zhu, Wenxia Xu, Lifeng Feng, Vivian Y Shin, Helen Morrison, Xian Wang, Hongchuan Jin
Heat shock factor 1 (HSF1) generally exhibits its properties under stress conditions. In tumors, HSF1 has a pleiotropic feature in regulating growth, survival, and aggressiveness of cancer cells. In this study, we found HSF1 was increased in colorectal cancer (CRC) and had a positive correlation with shorter disease-free survival (DFS). Knockdown of HSF1 in CRC cells attenuated their growth while inhibiting mTOR activation and glutamine metabolism. HSF1 inhibited the expression of microRNA137 (MIR137), which targeted GLS1 (glutaminase 1), thus stimulating GLS1 protein expression to promote glutaminolysis and mTOR activation...
April 14, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29735366/self-delivering-rnai-targeting-pd-1-improves-tumor-specific-t-cell-functionality-for-adoptive-cell-therapy-of-malignant-melanoma
#14
Maarten A Ligtenberg, Yago Pico de Coaña, Taisia Shmushkovich, Yuya Yoshimoto, Iva Truxova, Yuan Yang, Monica Betancur-Boissel, Alexey V Eliseev, Alexey D Wolfson, Rolf Kiessling
Adoptive cell therapy (ACT) is becoming a prominent alternative therapeutic treatment for cancer patients relapsing on traditional therapies. In parallel, antibodies targeting immune checkpoint molecules, such as cytotoxic-T-lymphocyte-associated antigen 4 (CTLA-4) and cell death protein 1 pathway (PD-1), are rapidly being approved for multiple cancer types, including as first line therapy for PD-L1-expressing non-small-cell lung cancer. The combination of ACT and checkpoint blockade could substantially boost the efficacy of ACT...
April 13, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29724686/hiv-specific-t-cells-generated-from-naive-t-cells-suppress-hiv-in-vitro-and-recognize-wide-epitope-breadths
#15
Shabnum Patel, Elizabeth Chorvinsky, Shuroug Albihani, Conrad Russell Cruz, R Brad Jones, Elizabeth J Shpall, David M Margolis, Richard F Ambinder, Catherine M Bollard
The Berlin Patient represents the first and only functional HIV cure achieved by hematopoietic stem cell transplant (HSCT). In subsequent efforts to replicate this result, HIV rebounded post-HSCT after withdrawal of antiretroviral therapy. Providing HIV-specific immunity through adoptive T cell therapy may prevent HIV rebound post-HSCT by eliminating newly infected cells before they can seed systemic infection. Adoptive T cell therapy has demonstrated success in boosting Epstein-Barr virus and cytomegalovirus-specific immunity post-HSCT, controlling viral reactivation...
April 12, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29735365/human-cd19-targeted-mouse-t-cells-induce-b-cell-aplasia-and-toxicity-in-human-cd19-transgenic-mice
#16
Christopher A Pennell, Jessie L Barnum, Cameron S McDonald-Hyman, Angela Panoskaltsis-Mortari, Megan J Riddle, Zhengming Xiong, Michael Loschi, Govindarajan Thangavelu, Heather M Campbell, Meghan D Storlie, Yosef Refaeli, Scott N Furlan, Michael C Jensen, Leslie S Kean, Jeffrey S Miller, Jakub Tolar, Mark J Osborn, Bruce R Blazar
The clinical success of chimeric antigen receptor (CAR) T cell therapy for CD19+ B cell malignancies can be limited by acute toxicities and immunoglobulin replacement needs due to B cell aplasia from persistent CAR T cells. Life-threatening complications include cytokine release syndrome and neurologic adverse events, the exact etiologies of which are unclear. To elucidate the underlying toxicity mechanisms and test potentially safer CAR T cells, we developed a mouse model in which human CD19 (hCD19)-specific mouse CAR T cells were adoptively transferred into mice whose normal B cells express a hCD19 transgene at hemizygous levels...
April 7, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29728295/gene-therapy-strategies-to-restore-er-proteostasis-in-disease
#17
REVIEW
Vicente Valenzuela, Kasey L Jackson, Sergio P Sardi, Claudio Hetz
Proteostasis alterations are proposed as a transversal hallmark of several pathological conditions, including metabolic disorders, mechanical injury, cardiac malfunction, neurodegeneration, and cancer. Strategies to improve proteostasis aim to reduce the accumulation of specific disease-related misfolded proteins or bolster the endogenous mechanisms to fold and degrade abnormal proteins. Endoplasmic reticulum (ER) stress is a common pathological signature of a variety of diseases, which engages the unfolded protein response (UPR) as a cellular reaction to mitigate ER stress...
April 7, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29724685/species-specific-involvement-of-integrin-%C3%AE-iib%C3%AE-3-in-a-monoclonal-antibody-ch12-triggers-off-target-thrombocytopenia-in-cynomolgus-monkeys
#18
Yiting Zhang, Jianhua Sun, Minjia Tan, Yongzhen Liu, Qian Li, Hua Jiang, Huamao Wang, Zonghai Li, Wei Wan, Hualiang Jiang, Henglei Lu, Bingshun Wang, Jin Ren, Likun Gong
CH12 is a novel humanized monoclonal antibody against epidermal growth factor receptor variant III (EGFRvIII) for cancer treatment. Unfortunately, in pre-clinical safety evaluation studies, acute thrombocytopenia was observed after administration of CH12 in cynomolgus monkeys, but not rats. More importantly, in vitro experiments found that CH12 can bind and activate platelets in cynomolgus monkey, but not human peripheral blood samples. Cynomolgus monkey-specific thrombocytopenia has been reported previously; however, the underlying mechanism remains unclear...
April 7, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29703700/improved-patency-of-eptfe-grafts-as-a-hemodialysis-access-site-by-seeding-autologous-endothelial-cells-expressing-fibulin-5-and-vegf
#19
Itai Tzchori, Mizied Falah, Denis Shteynberg, Dana Levin Ashkenazi, Zeev Loberman, Luba Perry, Moshe Y Flugelman
Small caliber synthetic vascular grafts used for dialysis access sites have high failure rates due to neointima formation and thrombosis. Seeding synthetic grafts with endothelial cells (ECs) provides a biocompatible surface that may prevent graft failure. We tested the use of ePTFE grafts seeded with autologous ECs expressing fibulin-5 and vascular endothelial growth factor (VEGF), as a dialysis access site in a porcine model. We connected the carotid arteries and jugular veins of 12 miniature pigs using 7-mm ePTFE grafts; five grafts were seeded with autologous venous ECs modified to express fibulin-5 and VEGF, and seven unseeded grafts were implanted at the same location and served as controls...
April 5, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29703699/oncolytic-virus-combination-therapy-killing-one-bird-with-two-stones
#20
REVIEW
Nikolas Tim Martin, John Cameron Bell
Over the last 60 years an eclectic collection of microbes has been tested in a variety of pre-clinical models as anti-cancer agents. At the forefront of this research are a number of virus-based platforms that have shown exciting activity in a variety of pre-clinical models and are collectively referred to as oncolytic viruses. Our true understanding of the potential and limitations of this therapeutic modality has been substantially advanced through clinical studies carried out over the last 25 years. Perhaps not surprising, as with all other cancer therapeutics, it has become clear that current oncolytic virus therapeutics on their own are unlikely to be effective in the majority of patients...
April 5, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
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