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 advisory board by O’Mahony Brian, Fletcher Simon, Baarslag Manuel, Khair Kate, Breederveld Daan, Jansen Nathalie, Pembroke Luke, Pinachyan Karen, Sivasubramaniyam Sujan

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The Relationship between the Antitumor Effect of the IL-12 Gene Therapy and the Expression of Th1 Cytokines in an HPV16-Positive Murine Tumor Model by Flor García Paz, Vicente Madrid Marina, Ausencio Morales Ortega, Abimelec Santander González, Oscar Peralta Zaragoza, Ana Burguete García, Kirvis Torres Poveda, José Moreno, Juan Alcocer González, Eva Hernandez Marquez, Victor Bermúdez Morales

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Addressing the limitations of the regulatory landscape in South Africa regarding advanced cell and gene therapies and related sectors involving human cells, tissues and organs by I M Viljoen, M S Pepper

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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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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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Cortical AAV-CNTF Gene Therapy Combined with Intraspinal Mesenchymal Precursor Cell Transplantation Promotes Functional and Morphological Outcomes after Spinal Cord Injury in Adult Rats by Stuart I. Hodgetts, Jun Han Yoon, Alysia Fogliani, Emmanuel A. Akinpelu, Danii Baron-Heeris, Imke G. J. Houwers, Lachlan P. G. Wheeler, Bernadette T. Majda, Sreya Santhakumar, Sarah J. Lovett, Emma Duce, Margaret A. Pollett, Tylie M. Wiseman, Brooke Fehily, Alan R. Harvey

“…Here we tested whether CNTF gene therapy targeting corticospinal neurons (CSN) in motor-related regions of the cerebral cortex promotes plasticity and regrowth of axons projecting into the female adult F344 rat spinal cord after moderate thoracic (T10) contusion injury (SCI). …”
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Endothelial and neuronal engagement by AAV-BR1 gene therapy alleviates neurological symptoms and lipid deposition in a mouse model of Niemann-Pick type C2 by Charlotte Laurfelt Munch Rasmussen, Signe Frost Frederiksen, Christian Würtz Heegaard, Maj Schneider Thomsen, Eva Hede, Bartosz Laczek, Jakob Körbelin, Daniel Wüstner, Louiza Bohn Thomsen, Markus Schwaninger, Ole N. Jensen, Torben Moos, Annette Burkhart

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<i>Ex Vivo</i> Regional Gene Therapy Compared to Recombinant BMP-2 for the Treatment of Critical-Size Bone Defects: An In Vivo Single-Cell RNA-Sequencing Study by Arijita Sarkar, Matthew C. Gallo, Jennifer A. Bell, Cory K. Mayfield, Jacob R. Ball, Mina Ayad, Elizabeth Lechtholz-Zey, Stephanie W. Chang, Osamu Sugiyama, Denis Evseenko, Jay R. Lieberman

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Genetic advancements in breast cancer treatment: a review by Marzieh Shokoohi, Sadaf Sedaghatshoar, Homaira Arian, Milad Mokarami, Fatemeh Habibi, Fatemeh Bamarinejad

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CRISPR-Cas9 in Cardiovascular Medicine: Unlocking New Potential for Treatment by Klaudia Bonowicz, Dominika Jerka, Klaudia Piekarska, Janet Olagbaju, Laura Stapleton, Munirat Shobowale, Andrzej Bartosiński, Magdalena Łapot, Yidong Bai, Maciej Gagat

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