Genetic Hearing Loss and Gene Therapy by Nathanial T Carpena, Min Young Lee

Subjects: “…gene therapy…”
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Adeno-associated virus-based gene therapy for hemophilia–addressing the gaps by Wolfgang Miesbach, Paul Batty, Pratima Chowdary, Sylvia Fong, Radoslaw Kaczmarek, Frank W.G. Leebeek, Brian Long, Johnny Mahlangu, Mike Makris, Glenn F. Pierce, Steven W. Pipe, Alok Srivastava, Jan Voorberg, Flora Peyvandi

Subjects: “…gene therapy…”
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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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Molecular cloning of EZH2 oncogene in chronic myeloid leukemia and therapeutic prospects by Juliana Costa Gaspar, Lucas Santos de Santana, Camila Menezes Freire de Souza, Marcos Montani Caseiro, Rosane Rezende de Souza Giuliani, Cleide Barbieri de Souza

Subjects: “…biotechnology. chronic myelogenous leukemia. molecular cloning. gene therapy.…”
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Local delivery of TGF-β1-mRNA decreases fibrosis in osteochondral defects by Gianluca Fontana, Brett Nemke, Yan Lu, Connie Chamberlain, Jae-Sung Lee, Joshua A. Choe, Hongli Jiao, Michael Nelson, Margot Amitrano, Wan-Ju Li, Mark Markel, William L. Murphy

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Living, Caring, Learning – The power of qualitative research in bleeding disorders care: One voice, from soloist to choir by Fletcher Simon

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A multi-stakeholder collaborative approach to awareness, education and support needed in the haemophilia gene therapy patient journey – a report on insights from a European patient... by O’Mahony Brian, Fletcher Simon, Baarslag Manuel, Khair Kate, Breederveld Daan, Jansen Nathalie, Pembroke Luke, Pinachyan Karen, Sivasubramaniyam Sujan

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Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives by Marco A. Luís, Marcelo A. D. Goes, Fátima Milhano Santos, Joana Mesquita, Paulo Tavares-Ratado, Cândida Teixeira Tomaz

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Recent advancements to enhance the therapeutic efficacy of antiepileptic drugs by Li Fuquan, Singh Akhilesh Vikram

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Brief summary of the regulatory frameworks of regenerative medicine therapies by Jinah Yoon, Sukhyang Lee, Sukhyang Lee, Min Jung Kim, Min Jung Kim, Jung-Hyun Kim, Jung-Hyun Kim

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THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES by Ugo Testa, Giuseppe Leone, Maria Domenica Cappellini

Subjects: “…Gene editing; gene therapy; Hemoglobinopathies; Thalassemia; Sickle Cell Anemia.…”
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Exploring patient and clinician perspectives on the benefits and risks of emerging therapies for the treatment of haemophilia: a qualitative study by Spoors John, Payne Katherine, Wright Stuart, Horsley Will, Bell Sadie, Cairns John

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Deciphering Oxidative Stress in Cardiovascular Disease Progression: A Blueprint for Mechanistic Understanding and Therapeutic Innovation by Zhaoshan Zhang, Jiawei Guo

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Current AAV-mediated gene therapy in sensorineural hearing loss by Jieyu Qi, Xiaolong Fu, Liyan Zhang, Fangzhi Tan, Nianci Li, Qiuhan Sun, Xiaojie Hu, Zuhong He, Ming Xia, Renjie Chai

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From bench to bedside: Developing CRISPR/Cas-based therapy for ocular diseases by Qing Zhao, Linxin Wei, Youxin Chen

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A systematic screening assay identifies efficient small guide RNAs for CRISPR activation by Elin Arvidsson, Diana Duarte Lobo, Diana Duarte Lobo, Ermelinda Sabarese, Fabio Duarte, Rui Jorge Nobre, Rui Jorge Nobre, Rui Jorge Nobre, Luis Quintino, Cecilia Lundberg

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AAV-based vectors for human diseases modeling in laboratory animals by Timur I. Aliev, Dmitry V. Yudkin

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Recombinant AAV batch profiling by nanopore sequencing elucidates product-related DNA impurities and vector genome length distribution by Florian Dunker-Seidler, Kathrin Breunig, Magdalena Haubner, Florian Sonntag, Markus Hörer, Rebecca C. Feiner

Subjects: “…nanopore sequencing, gene therapy…”
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