Gene therapy by hepatocyte growth factor results in regression of experimental liver fibrosis by N. A. Dzhoyashvili, N. I. Kalinina, I. B. Beloglazova, Z. I. Tsokolayeva, P. I. Makarevich, Yu. L. Perov, Ye. V. Parfenova, V. A. Tkachuk

“…Obtained results allow to consider gene therapy by hepatocyte growth factor in composition of non-viral vectors as one of perspective methods of treatment of chronic liver diseases.…”
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The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia by Ekati Drakopoulou, Eleni Papanikolaou, Nicholas P. Anagnou

“…The major breakthrough in β-thalassemia gene therapy occurred a decade ago with the development of globin LVs. …”
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Gene Therapy: A step toward advanced orthodontics – A narrative review by Monika Shukla, Shalu Jain, C. Munish Reddy, Pradeep Raghav, Kumar Amit, Rinchin Yangzom, Shivika Garg, Aastha Kamrani

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Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations by Lucas Tricoli, Sunetra Sase, Julia L. Hacker, Vi Pham, Maxwell Chappell, Laura Breda, Stephanie N. Hurwitz, Naoto Tanaka, Carlo Castruccio Castracani, Amaliris Guerra, Zhongqi Hou, Lars Schlotawa, Karthikeyan Radhakrishnan, Matthew Hogenauer, Aoife Roche, John Everett, Frederic Bushman, Peter Kurre, Rebecca Ahrens-Nicklas, Laura A. Adang, Adeline L. Vanderver, Stefano Rivella

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Current views on etiology, diagnosis, epidemiology and gene therapy of maturity onset diabetes in the young by Lilya U. Dzhemileva, Elena N. Zakharova, Anna O. Goncharenko, Maria V. Vorontsova, S. A. Rumyantsev, Natalia G. Mokrysheva, Marina Y. Loguinova, Vladimir P. Chekhonin

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Antigen-Specific Gene Therapy after Immunisation Reduces the Severity of Collagen-Induced Arthritis by Tove Eneljung, Sara Tengvall, Pernilla Jirholt, Louise Henningsson, Rikard Holmdahl, Kenth Gustafsson, Inger Gjertsson

“…Thus, antigen-specific gene therapy is effective when administered throughout the inflammatory course of arthritis and offers a good model for investigation of the basic mechanisms during tolerance in CIA.…”
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Ensuring patient access to gene therapies for rare diseases: Navigating reimbursement and coverage challenges by Diane Berry, Carolyn Hickey, Lisa Kahlman, James Long, Christina Markus, Caitlin K. McCombs

“…It emphasizes the need to account for the broader value of gene therapies, incorporate input from disease-specific clinical experts in payer coverage decisions, and reduce administrative barriers to coverage. …”
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The Multifaceted Role of Extracellular Vesicles in Glioblastoma: microRNA Nanocarriers for Disease Progression and Gene Therapy by Natalia Simionescu, Radu Zonda, Anca Roxana Petrovici, Adriana Georgescu

“…In this review, we discuss the role of EVs and their miRNA content in GB progression and resistance to therapy, with emphasis on their potential as diagnostic, prognostic and disease monitoring biomarkers and as nanocarriers for gene therapy.…”
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HMGB1 Box A gene therapy to alleviate bleomycin-induced pulmonary fibrosis in rats by Rathasapa Patarat, Suchanart Chuaybudda, Sakawdaurn Yasom, Apiwat Mutirangura

“…Previously, a new gene therapy technology using Box A of the HMGB1 plasmid (Box A) was used to reverse cellular senescence and cure liver fibrosis in aged rats. …”
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Oral Tolerance: A New Tool for the Treatment of Gastrointestinal Inflammatory Disorders and Liver-Directed Gene Therapy by Yaron Ilan

“…The use of oral tolerance toward adenoviruses and colitis-extracted proteins for long term gene therapy and alleviation of experimental colitis, and the mechanisms of tolerance induction are presented. …”
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Cardiac Gene Therapy With Phosphodiesterase 2A Limits Remodeling and Arrhythmias in Mouse Models of Heart Failure by Rima Kamel, Aurélia Bourcier, Jean Piero Margaria, Valentin Jin, Audrey Varin, Agnès Hivonnait, Françoise Mercier‐Nomé, Delphine Mika, Alessandra Ghigo, Flavien Charpentier, Vincent Algalarrondo, Emilio Hirsch, Rodolphe Fischmeister, Grégoire Vandecasteele, Jérôme Leroy

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Gene Therapy with Endogenous Inhibitors of Angiogenesis for Neovascular Age-Related Macular Degeneration: Beyond Anti-VEGF Therapy by Selwyn M. Prea, Elsa C. Chan, Gregory J. Dusting, Algis J. Vingrys, Bang V. Bui, Guei-Sheung Liu

“…In this mini review, we summarize current theories pertaining to the application of gene therapy for neovascular AMD and the potential benefits when used in conjunction with endogenous antiangiogenic proteins.…”
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Secreted PD-L1 alleviates inflammatory arthritis in mice through local and systemic AAV gene therapy by Wenjun Li, Wenjun Li, Junjiang Sun, Susi Feng, Ariana La Rosa, Panli Zhang, Eveline Y. Wu, Richard Loeser, Chengwen Li, Chengwen Li, Chengwen Li

“…To study the therapeutic effect of systemic expression of sPD-L1, we administered AAV8/shPD-L1 gene therapy in CIA mice via retro-orbital injection and found significant improvements in joint inflammation and paw swelling, exhibiting similar phenotypes to that in naïve mice. …”
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Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy by Peter Gee, Huaigeng Xu, Akitsu Hotta

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Lentivirus‐mediated gene therapy for Fabry disease: 5‐year End‐of‐Study results from the Canadian FACTs trial by Aneal Khan, Dwayne L. Barber, William M. McKillop, C. Anthony Rupar, Christiane Auray‐Blais, Graeme Fraser, Daniel H. Fowler, Alexandra Berger, Ronan Foley, Armand Keating, Michael L. West, Jeffrey A. Medin

“…Highlights This was the first gene therapy clinical trial to be completed for Fabry disease. …”
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Subthalamic hGAD65 Gene Therapy and Striatum TH Gene Transfer in a Parkinson’s Disease Rat Model by Deyu Zheng, Xiaohua Jiang, Junpeng Zhao, Deyi Duan, Huanying Zhao, Qunyuan Xu

“…The aim of the present study is to detect a combination method to utilize gene therapy for the treatment of Parkinson’s disease (PD). …”
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E-Selectin/AAV Gene Therapy Promotes Myogenesis and Skeletal Muscle Recovery in a Mouse Hindlimb Ischemia Model by Antoine J. Ribieras, Yulexi Y. Ortiz, Yan Li, Nga T. Le, Carlos T. Huerta, Francesca A. Voza, Hongwei Shao, Roberto I. Vazquez-Padron, Zhao-Jun Liu, Omaida C. Velazquez

“…These results suggest a potential role for E-sel/AAV gene therapy as a nonsurgical adjunct in patients with life-limiting PAD.…”
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Mathematical modeling of glioma therapy using oncolytic viruses by Baba Issa Camara, Houda Mokrani, Evans K. Afenya

Subjects: “…cancer gene therapy…”
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Engineering a bacterial toxin deaminase from the DYW-family into a novel cytosine base editor for plants and mammalian cells by Dingbo Zhang, Fiona Parth, Laura Matos da Silva, Teng-Cheong Ha, Axel Schambach, Jens Boch

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Therapeutics in paediatric genetic diseases: current and future landscape by Ai Ling Koh, Saumya Shekhar Jamuar

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