Search Results - "Molecular therapy"

Current Clinical Applications of In Vivo Gene Therapy with AAVs by Mendell, Jerry R., Al-Zaidy, Samiah A., Rodino-Klapac, Louise R., Goodspeed, Kimberly, Gray, Steven J., Kay, Christine N., Boye, Sanford L., Boye, Shannon E., George, Lindsey A., Salabarria, Stephanie, Corti, Manuela, Byrne, Barry J., Tremblay, Jacques P.

“…Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases affect over 30 million Americans. For more than 30 years, hundreds of…”
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Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery by Kowalski, Piotr S., Rudra, Arnab, Miao, Lei, Anderson, Daniel G.

“…mRNA has broad potential as a therapeutic. Current clinical efforts are focused on vaccination, protein replacement therapies, and treatment of genetic…”
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Immune Responses to Viral Gene Therapy Vectors by Shirley, Jamie L., de Jong, Ype P., Terhorst, Cox, Herzog, Roland W.

“…Several viral vector-based gene therapy drugs have now received marketing approval. A much larger number of additional viral vectors are in various stages of…”
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AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer by Costa Verdera, Helena, Kuranda, Klaudia, Mingozzi, Federico

“…Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-term efficacy in a number of trials across target organs, including…”
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circRNA_0025202 Regulates Tamoxifen Sensitivity and Tumor Progression via Regulating the miR-182-5p/FOXO3a Axis in Breast Cancer by Sang, Yuting, Chen, Bing, Song, Xiaojin, Li, Yaming, Liang, Yiran, Han, Dianwen, Zhang, Ning, Zhang, Hanwen, Liu, Ying, Chen, Tong, Li, Chen, Wang, Lijuan, Zhao, Wenjing, Yang, Qifeng

“…Tamoxifen is the most commonly used endocrine therapy for patients with hormone receptor (HR)-positive breast cancer. Despite its initial therapeutic efficacy,…”
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Lipid Nanoparticle Systems for Enabling Gene Therapies by Cullis, Pieter R., Hope, Michael J.

“…Genetic drugs such as small interfering RNA (siRNA), mRNA, or plasmid DNA provide potential gene therapies to treat most diseases by silencing pathological…”
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Dominant-Negative TGF-β Receptor Enhances PSMA-Targeted Human CAR T Cell Proliferation And Augments Prostate Cancer Eradication by Kloss, Christopher C., Lee, Jihyun, Zhang, Aaron, Chen, Fang, Melenhorst, Jan Joseph, Lacey, Simon F., Maus, Marcela V., Fraietta, Joseph A., Zhao, Yangbing, June, Carl H.

“…Cancer has an impressive ability to evolve multiple processes to evade therapies. While immunotherapies and vaccines have shown great promise, particularly in…”
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mRNA as a Transformative Technology for Vaccine Development to Control Infectious Diseases by Maruggi, Giulietta, Zhang, Cuiling, Li, Junwei, Ulmer, Jeffrey B., Yu, Dong

“…In the last two decades, there has been growing interest in mRNA-based technology for the development of prophylactic vaccines against infectious diseases…”
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CAR T Cell Therapy for Solid Tumors: Bright Future or Dark Reality? by Wagner, Jessica, Wickman, Elizabeth, DeRenzo, Christopher, Gottschalk, Stephen

“…Chimeric antigen receptor (CAR) T cell therapy has garnered significant excitement due to its success for hematological malignancies in clinical studies…”
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Exosomes Produced from 3D Cultures of MSCs by Tangential Flow Filtration Show Higher Yield and Improved Activity by Haraszti, Reka Agnes, Miller, Rachael, Stoppato, Matteo, Sere, Yves Y., Coles, Andrew, Didiot, Marie-Cecile, Wollacott, Rachel, Sapp, Ellen, Dubuke, Michelle L., Li, Xuni, Shaffer, Scott A., DiFiglia, Marian, Wang, Yang, Aronin, Neil, Khvorova, Anastasia

“…Exosomes can deliver therapeutic RNAs to neurons. The composition and the safety profile of exosomes depend on the type of the exosome-producing cell…”
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Preclinical and Clinical Demonstration of Immunogenicity by mRNA Vaccines against H10N8 and H7N9 Influenza Viruses by Bahl, Kapil, Senn, Joe J., Yuzhakov, Olga, Bulychev, Alex, Brito, Luis A., Hassett, Kimberly J., Laska, Michael E., Smith, Mike, Almarsson, Örn, Thompson, James, Ribeiro, Amilcar (Mick), Watson, Mike, Zaks, Tal, Ciaramella, Giuseppe

“…Recently, the World Health Organization confirmed 120 new human cases of avian H7N9 influenza in China resulting in 37 deaths, highlighting the concern for a…”
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Emerging mechanisms and targeted therapy of ferroptosis in cancer by Wang, Haiyan, Cheng, Yan, Mao, Chao, Liu, Shuang, Xiao, Desheng, Huang, Jun, Tao, Yongguang

“…Ferroptosis is an iron- and lipid reactive oxygen species (ROS)-dependent form of programmed cell death that is distinct from other forms of regulatory cell…”
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COVID-19 mRNA vaccines: Platforms and current developments by Szabó, Gábor Tamás, Mahiny, Azita Josefine, Vlatkovic, Irena

“…Since the first successful application of messenger ribonucleic acid (mRNA) as a vaccine agent in a preclinical study nearly 30 years ago, numerous advances…”
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Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy by Duan, Dongsheng

“…Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene mutation. Conceptually, replacing the mutated gene with a normal one…”
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ncRNA-Encoded Peptides or Proteins and Cancer by Wang, Jizhong, Zhu, Song, Meng, Nan, He, Yutian, Lu, Ruixun, Yan, Guang-Rong

“…Non-coding RNAs (ncRNAs) are unique RNA transcripts that have been widely identified in the eukaryotic genome and have been shown to play key roles in the…”
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A versatile platform for generating engineered extracellular vesicles with defined therapeutic properties by Dooley, Kevin, McConnell, Russell E., Xu, Ke, Lewis, Nuruddeen D., Haupt, Sonya, Youniss, Madeleine R., Martin, Shelly, Sia, Chang Ling, McCoy, Christine, Moniz, Raymond J., Burenkova, Olga, Sanchez-Salazar, Jorge, Jang, Su Chul, Choi, Bryan, Harrison, Rane A., Houde, Damian, Burzyn, Dalia, Leng, Charan, Kirwin, Katherine, Ross, Nikki L., Finn, Jonathan D., Gaidukov, Leonid, Economides, Kyriakos D., Estes, Scott, Thornton, James E., Kulman, John D., Sathyanarayanan, Sriram, Williams, Douglas E.

“…Extracellular vesicles (EVs) are an important intercellular communication system facilitating the transfer of macromolecules between cells. Delivery of…”
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Phase I Study of Lentiviral-Transduced Chimeric Antigen Receptor-Modified T Cells Recognizing Mesothelin in Advanced Solid Cancers by Haas, Andrew R., Tanyi, Janos L., O’Hara, Mark H., Gladney, Whitney L., Lacey, Simon F., Torigian, Drew A., Soulen, Michael C., Tian, Lifeng, McGarvey, Maureen, Nelson, Anne Marie, Farabaugh, Caitlin S., Moon, Edmund, Levine, Bruce L., Melenhorst, J. Joseph, Plesa, Gabriela, June, Carl H., Albelda, Steven M., Beatty, Gregory L.

“…This phase I study investigated the safety and activity of lentiviral-transduced chimeric antigen receptor (CAR)-modified autologous T cells redirected against…”
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M2 Macrophage-Derived Exosomes Promote Angiogenesis and Growth of Pancreatic Ductal Adenocarcinoma by Targeting E2F2 by Yang, Yuhan, Guo, Zengya, Chen, Weiwei, Wang, Xiaofeng, Cao, Meng, Han, Xuan, Zhang, Kundong, Teng, Buwei, Cao, Jun, Wu, Weidong, Cao, Peng, Huang, Chen, Qiu, Zhengjun

“…Pancreatic ductal adenocarcinoma (PDAC), one of the most aggressive tumors all over the world, has a generally poor prognosis, and its progression is…”
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CAR T Cells Administered in Combination with Lymphodepletion and PD-1 Inhibition to Patients with Neuroblastoma by Heczey, Andras, Louis, Chrystal U., Savoldo, Barbara, Dakhova, Olga, Durett, April, Grilley, Bambi, Liu, Hao, Wu, Mengfeng F., Mei, Zhuyong, Gee, Adrian, Mehta, Birju, Zhang, Huimin, Mahmood, Nadia, Tashiro, Haruko, Heslop, Helen E., Dotti, Gianpietro, Rooney, Cliona M., Brenner, Malcolm K.

“…Targeting disialoganglioside (GD2) on neuroblastoma (NB) with T cells expressing a first-generation chimeric antigen receptor (CAR) was safe, but the cells had…”
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Role of exosomal non-coding RNAs from tumor cells and tumor-associated macrophages in the tumor microenvironment by Xu, Zijie, Chen, Yi, Ma, Ling, Chen, Yizhang, Liu, Jingya, Guo, Yuchen, Yu, Ting, Zhang, Lianghui, Zhu, Lingjun, Shu, Yongqian

“…Exosomes have a crucial role in intercellular communication and mediate interactions between tumor cells and tumor-associated macrophages (TAMs)…”
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