Results 41 to 50 of about 2,049,710 (211)

Macrophage Transcriptomic Alterations Driven by Alphavirus-Based Cancer Immunotherapy Vectors

Journal of Immunology Research
Cancer cells promote the polarisation of tumour-associated macrophages (TAMs) into pro-tumorigenic M2-like phenotype, contributing to cancer progression. Reprogramming TAMs by viral immunotherapy vectors represents a promising strategy for cancer therapy.
Ksenija Korotkaja, Darija Lapina, Zhanna Rudevica, Anna Zajakina   +3 more
doaj   +1 more source

Urocortin 2 Gene Transfer Improves Glycemic Control and Reduces Retinopathy and Mortality in Murine Insulin Deficiency. [PDF]

, 2020
Type 1 diabetes affects 20 million patients worldwide. Insulin is the primary and commonly the sole therapy for type 1 diabetes. However, only a minority of patients attain the targeted glucose control and reduced adverse events.
Bhargava, Raag, Gao, Mei Hua, Giamouridis, Dimosthenis, Guo, Tracy, Hammond, H Kirk, Huu, Viet Anh Nguyen, Kim, Young Chul, Lai, N Chin, Lantier, Louise, Skowronska-Krawczyk, Dorota, Xia, Bing   +10 more
core  

Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor

EMBO Molecular Medicine
Physiological regulation of transgene expression is a major challenge in gene therapy. Onasemnogene abeparvovec (Zolgensma®) is an approved adeno-associated virus (AAV) vector gene therapy for infants with spinal muscular atrophy (SMA), however, adverse ...
Qing Xie, Xiupeng Chen, Hong Ma, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Gerald F Cox, Fiona Weaver, Jun Yang, Zachary Kennedy, Alisha Gruntman, Ailing Du, Qin Su, Ran He, Phillip WL Tai, Guangping Gao, Jun Xie   +16 more
doaj   +1 more source

Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes

Molecular Therapy: Methods & Clinical Development, 2018
The generation of clinical good manufacturing practices (GMP)-grade adeno-associated virus (AAV) vectors requires purification strategies that support the generation of vectors of high purity, and that exhibit a good safety and efficacy profile. To date,
Shelley A. Nass, Maryellen A. Mattingly, Denise A. Woodcock, Brenda L. Burnham, Jeffrey A. Ardinger, Shayla E. Osmond, Amy M. Frederick, Abraham Scaria, Seng H. Cheng, Catherine R. O’Riordan   +9 more
doaj   +1 more source

The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis [PDF]

, 2012
This article is available open access through the publisher’s website at the link below. Copyright @ 2013 The American Society of Gene & Cell Therapy.Genotoxicity models are extremely important to assess retroviral vector biosafety before gene therapy ...
Al-Allaf, F, Andrews, R, Arens, A, Bigger, B, Buckley, SM, Cheung, WT, Coutelle, C, Dighe, N, Gregory, L, Holder, M, Li, T, Montini, E, Newbold, RF, Nowrouzi, A, Osejindu, E, Paruzynski, A, Reja, S, Roberts, TP, Ruthe, A, Schmidt, M, Themis, M, Themis, M, Thrasher, AJ, von Kalle, C, Waddington, SN, Wang, Y, Zhang, X   +26 more
core   +1 more source

Establishing the cost of implementing a performance-based, managed entry agreement for a hypothetical CAR T-cell therapy

Journal of Market Access & Health Policy, 2018
Background: Market access stakeholders consider the adoption of Managed Entry Agreements (MEAs), however a clearly described methodology to quantify their implementation burden is not available in the public domain.
Panos Kefalas, Omar Ali, Jesper Jørgensen, Nick Merryfield, Tim Richardson, Adam Meads, Laura Mungapen, Matthew Durdy   +7 more
doaj   +1 more source

Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model

Molecular Therapy: Methods & Clinical Development, 2023
Limb-girdle muscular dystrophy (LGMD) type 2C/R5 results from mutations in the γ-sarcoglycan (SGCG) gene and is characterized by muscle weakness and progressive wasting.
Young-Eun Seo, Stephen H. Baine, Amber N. Kempton, Oliver C. Rogers, Sarah Lewis, Kaitlin Adegboye, Alex Haile, Danielle A. Griffin, Ellyn L. Peterson, Eric R. Pozsgai, Rachael A. Potter, Louise R. Rodino-Klapac   +11 more
doaj   +1 more source

Genome-Wide Association Study and Gene Expression Analysis Identifies CD84 as a Predictor of Response to Etanercept Therapy in Rheumatoid Arthritis [PDF]

, 2013
Anti-tumor necrosis factor alpha (anti-TNF) biologic therapy is a widely used treatment for rheumatoid arthritis (RA). It is unknown why some RA patients fail to respond adequately to anti-TNF therapy, which limits the development of clinical biomarkers ...
A Parker, AI Catrina, AL Price, Alison Motsinger-Reif, AM van Gestel, Ann W. Morgan, Anne Barton, Anthony G. Wilson, Atsuo Taniguchi, B Devlin, Barbara E. Stranger, BE Stranger, BJ Scallon, C Liu, C Miceli-Richard, Chikashi Terao, Corinne Miceli, Cornelia F. Allaart, D Aeberli, D Plant, D Plant, DL Scott, Dorothee Diogo, EA Stahl, EA Stahl, EJ Toonen, Eli A. Stahl, Elizabeth W. Karlson, FM Batliwalla, Fumihiko Matsuda, Gert Jan Wolbink, GM Cooper, Gosia Trynka, H Canhao, Helena Canhao, Henk-Jan Guchelaar, Hisashi Yamanaka, IB McInnes, Irene E. van der Horst-Bruinsma, J Agnholt, J Cui, J Ernst, J Marchini, J. Bart A. Crusius, Jing Cui, Johan Askling, John D. Isaacs, João Eurico Fonseca, Katsunori Ikari, Kimme L. Hyrich, Koichiro Ohmura, L Klareskog, L Padyukov, Larry W. Moreland, LD Ward, Leonid Padyukov, Lindsey A. Criswell, M Martin, Manik Kuchroo, Marieke E. Doorenspleet, Marieke Herenius, Marieke J. H. Coenen, Mart van de Laar, Maša Umiċeviċ Mirkov, Michael E. Weinblatt, MJ Coenen, ML Prevoo, MM Soliman, Namrata Gupta, Nancy A. Shadick, Niek de Vries, O Stegle, Paul-Peter Tak, Peter K. Gregersen, Philip L. De Jager, PI de Bakker, Piet L. C. M. van Riel, PL De Jager, R Prajapati, Rene E. M. Toes, RM Plenge, Robert M. Plenge, Robert P. Kimberly, S Gudbrandsdottir, S Purcell, S. Louis Bridges, SA Gauthier, Saedis Saevarsdottir, Sara Wedrén, SG Tangye, Shigeki Momohara, Soumya Raychaudhuri, Tom W. J. Huizinga, Towfique Raj, Tsuneyo Mimori, Xavier Mariette, Y Okada, Yukinori Okada   +97 more
core   +8 more sources

Surface immobilization of hexa-histidine-tagged adeno-associated viral vectors for localized gene delivery. [PDF]

, 2010
Adeno-associated viral (AAV) vectors, which are undergoing broad exploration in clinical trials, have significant promise for therapeutic gene delivery because of their safety and delivery efficiency.
Bethi, SR, Gujraty, K, Jang, J-H, Kane, RS, Koerber, JT, Schaffer, DV   +5 more
core   +5 more sources

Application of Raman Spectroscopy and Univariate Modelling As a Process Analytical Technology for Cell Therapy Bioprocessing

Frontiers in Medicine, 2018
Cell therapies offer unquestionable promises for the treatment, and in some cases even the cure, of complex diseases. As we start to see more of these therapies gaining market authorization, attention is turning to the bioprocesses used for their ...
Marc-Olivier Baradez, Daniela Biziato, Enas Hassan, Damian Marshall   +3 more
doaj   +1 more source