Protocol for a high titer of BaEV-Rless pseudotyped lentiviral vector: Focus on syncytium formation and detachment von Noguchi, Kazuhiro, Ikawa, Yasuhiro, Takenaka, Mika, Sakai, Yuta, Fujiki, Toshihiro, Kuroda, Rie, Chappell, Maxwell, Ghiaccio, Valentina, Rivella, Stefano, Wada, Taizo

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CURING HEMOGLOBINOPATHIES: CHALLENGES AND ADVANCES OF CONVENTIONAL AND NEW GENE THERAPY APPROACHES von Motta, Irene, Ghiaccio, Valentina, Cosentino, Andrea, Breda, Laura

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Inclusion of a short hairpin RNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin von Pires Lourenco, Silvia, Jarocha, Danuta, Ghiaccio, Valentina, Guerra, Amaliris, Abdulmalik, Osheiza, La, Ping, Zezulin, Alexandra, Smith-Whitley, Kim, Kwiatkowski, Janet L., Guzikowski, Virginia, Nakamura, Yukio, Raabe, Tobias, Breda, Laura, Rivella, Stefano

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2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance von Dong, Alisa, Ghiaccio, Valentina, Motta, Irene, Guo, Shuling, Peralta, Raechel, Freier, Susan M, Watt, Andy, Damle, Sagar, Ikawa, Yasuhiro, Jarocha, Danuta, Chappell, Maxwell, Stephanou, Coralea, Delbini, Paola, Chen, Connie, Christou, Soteroula, Kleanthous, Marina, Smith-Whitley, Kim, Manwani, Deepa, Casu, Carla, Abdulmalik, Osheiza, Cappellini, Maria Domenica, Rivella, Stefano, Breda, Laura

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Establishment of High Titer Baboon Endogenous Retrovirus Envelope Pseudotyped Lentiviral Vector Production Protocol Focusing on Syncytium Formation and Exfoliation von Noguchi, Kazuhiro, Ikawa, Yasuhiro, Takenaka, Mika, Sakai, Yuta, Fujiki, Toshihiro, Kuroda, Rie, Chappell, Maxwell, Ghiaccio, Valentina, Rivella, Stefano, Wada, Taizo

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An Orchestrated Balance between Mitochondria Biogenesis, Iron-Sulfur Cluster Synthesis and Cellular Iron Acquisition von La, Ping, Ghiaccio, Valentina, Zhang, Jianbing, Rivella, Stefano

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Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges von Ghiaccio, Valentina, Chappell, Maxwell, Rivella, Stefano, Breda, Laura

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Mitochondria Biogenesis Modulates Iron-Sulfur Cluster Synthesis to Increase Cellular Iron Uptake von La, Ping, Oved, Joseph H, Ghiaccio, Valentina, Rivella, Stefano

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A Severe Mouse Model of Alpha-Thalassemia to Study Abnormal Iron Metabolism and Erythropoiesis, Hematopoietic Stem Cell Behavior and Development of a Gene Therapy Approach for Its... von Chappell, Maxwell, Jarocha, Danuta Jadwiga, Breda, Laura, Ghiaccio, Valentina, Triebwasser, Michael, Fedorky, Megan, Guerra, Amaliris, Gollomp, Kandace, Teawtrakul, Nattiya, Glentis, Stavros, Kattamis, Antonis, Abdulmalik, Osheiza, Rivella, Stefano

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Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-Thalassemia von Chappell, Maxwell, Breda, Laura, Jarocha, Danuta Jadwiga, Triebwasser, Michael, Papp, Tyler E, Ghiaccio, Valentina, Fedorky, Megan T, Guerra, Amaliris, Gollomp, Kandace, Abdulmalik, Osheiza Y., Teawtrakul, Nattiya, Glentis, Stavros, Kattamis, Antonis, Parhiz, Hamideh, Rivella, Stefano

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Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies von Breda, Laura, Ghiaccio, Valentina, Pires Lourenco, Silvia, Jarocha, Danuta Jadwiga, Ikawa, Yasuhiro, Tanaka, Naoto, Guzikowski, Virginia, Casu, Carla, Smith-Whitley, Kim, Kwiatkowski, Janet L., Rivella, Stefano

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Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies von Breda, Laura, Ghiaccio, Valentina, Tanaka, Naoto, Jarocha, Danuta, Ikawa, Yasuhiro, Abdulmalik, Osheiza, Dong, Alisa, Casu, Carla, Raabe, Tobias D., Shan, Xiaochuan, Danet-Desnoyers, Gwenn A., Doto, Aoife M., Everett, John, Bushman, Frederic D., Radaelli, Enrico, Assenmacher, Charles A., Tarrant, James C., Hoepp, Natalie, Kurita, Ryo, Nakamura, Yukio, Guzikowski, Virginia, Smith-Whitley, Kim, Kwiatkowski, Janet L., Rivella, Stefano

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Inclusion of a short hairpin RNA targeting BCL11A into a b-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin von Lourenco, Silvia Pires, Jarocha, Danuta, Ghiaccio, Valentina, Guerra, Amaliris, Abdulmalik, Osheiza, La, Ping, Zezulin, Alexandra, Smith-Whitley, Kim, Kwiatkowski, Janet L., Guzikowski, Virginia, Nakamura, Yukio, Raabe, Tobias, Breda, Laura, Rivella, Stefano

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Improved Lentiviral Vectors for the Cure of Hemoglobinopathies von Breda, Laura, Ghiaccio, Valentina, Zaidi, Hanyia, Pires Lourenco, Silvia, Casu, Carla, Smith-Whitley, Kim, Kwiatkowski, Janet L., Carrion, Vanessa, Guzikowski, Virginia, Kurita, Ryo, Nakamura, Yukio, Rivella, Stefano

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PP-14, a Novel Structurally-Enhanced Antisickling Allosteric Hemoglobin Effector, Increases Oxygen Affinity and Disrupts Hemoglobin S Polymer Formation von Safo, Martin K., Pagare, Piyusha P, Ghatge, Mohini, Casu, Carla, Ghiaccio, Valentina, Anabaraonye, Nancy, Xu, Xiaomeng, Rivella, Stefano, Hines, Patrick C., Liu, Ke, Zhang, Yan, Venitz, Jürgen, Abdulmalik, Osheiza

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2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring hemoglobin A production and chain rebalance von Dong, Alisa, Ghiaccio, Valentina, Motta, Irene, Guo, Shuling, Peralta, Raechel, Freier, Susan M., Watt, Andy, Damle, Sagar, Ikawa, Yasuhiro, Jarocha, Danuta, Chappell, Maxwell, Stephanou, Coralea, Delbini, Paola, Chen, Connie, Christou, Soteroula, Kleanthous, Marina, Smith-Whitley, Kim, Manwani, Deepa, Casu, Carla, Abdulmalik, Osheiza, Cappellini, Maria Domenica, Rivella, Stefano, Breda, Laura

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Tumor Microenvironment Remodeling by 4-Methylumbelliferone Boosts the Antitumor Effect of Combined Immunotherapy in Murine Colorectal Carcinoma von Malvicini, Mariana, Fiore, Esteban, Ghiaccio, Valentina, Piccioni, Flavia, Rizzo, Miguel, Olmedo Bonadeo, Lucila, García, Mariana, Rodríguez, Marcelo, Bayo, Juan, Peixoto, Estanislao, Atorrasagasti, Catalina, Alaniz, Laura, Aquino, Jorge, Matar, Pablo, Mazzolini, Guillermo

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2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies von Ghiaccio, Valentina, Dong, Alisa, Motta, Irene, Guo, Shuling, Peralta, Raechel, Freier, Susan M, Watt, Andy, Ikawa, Yasuhiro, Chappell, Maxwell, Stephanou, Coralea, Delbini, Paola, Chen, Connie, Christou, Soteroula, Kleanthous, Marina, Smith-Whitley, Kim, Manwani, Deepa, Casu, Carla, Cappellini, Maria Domenica, Abdulmalik, Osheiza, Rivella, Stefano, Breda, Laura

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Adult Hemoglobin Production, Chain Rebalance, and Splice Correction in IVS2-745 Beta-Thalassemia Patient Cells Using 2'-O-Methoxyethyl Splice-Switching Oligos von Dong, Alisa Cheung, Ghiaccio, Valentina, Motta, Irene, Guo, Shuling, Peralta, Raechel, Stephanou, Coralea, Delbini, Paola, Chen, Connie, Christou, Soteroula, Kleanthous, Marina, Cappellini, Maria Domenica, Abdulmalik, Osheiza, Breda, Laura, Rivella, Stefano

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Abstract B178: 4-methylumbelliferone modulates tumor microenvironment improving the antitumor efficacy of combined gene-based immunotherapy in murine colon adenocarcinoma von Malvicini, Mariana, Fiore, Esteban, Ghiaccio, Valentina, Piccioni, Flavia, Rizzo, Miguel, Garcia, Mariana, Rodriguez, Marcelo, Bayo, Juan, Peixoto, Estanislao, Atorrasagasti, Catalina, Alaniz, Laura, Matar, Pablo, Mazzolini, Guillermo

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