Protocol for a high titer of BaEV-Rless pseudotyped lentiviral vector: Focus on syncytium formation and detachment
Noguchi, Kazuhiro, Ikawa, Yasuhiro, Takenaka, Mika, Sakai, Yuta, Fujiki, Toshihiro, Kuroda, Rie, Chappell, Maxwell, Ghiaccio, Valentina, Rivella, Stefano, Wada, Taizo
Published in Journal of virological methods (01.04.2023)
Published in Journal of virological methods (01.04.2023)
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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
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
Published in Haematologica (Roma) (01.10.2021)
Published in Haematologica (Roma) (01.10.2021)
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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
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
Published in Haematologica (Roma) (01.05.2019)
Published in Haematologica (Roma) (01.05.2019)
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Establishment of High Titer Baboon Endogenous Retrovirus Envelope Pseudotyped Lentiviral Vector Production Protocol Focusing on Syncytium Formation and Exfoliation
Noguchi, Kazuhiro, Ikawa, Yasuhiro, Takenaka, Mika, Sakai, Yuta, Fujiki, Toshihiro, Kuroda, Rie, Chappell, Maxwell, Ghiaccio, Valentina, Rivella, Stefano, Wada, Taizo
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges
Ghiaccio, Valentina, Chappell, Maxwell, Rivella, Stefano, Breda, Laura
Published in Molecular diagnosis & therapy (01.04.2019)
Published in Molecular diagnosis & therapy (01.04.2019)
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Mitochondria Biogenesis Modulates Iron-Sulfur Cluster Synthesis to Increase Cellular Iron Uptake
La, Ping, Oved, Joseph H, Ghiaccio, Valentina, Rivella, Stefano
Published in DNA and cell biology (01.05.2020)
Published in DNA and cell biology (01.05.2020)
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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 Treatment
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
Published in Blood (23.11.2021)
Published in Blood (23.11.2021)
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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
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
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy
Chappell, Maxwell E., Breda, Laura, Tricoli, Lucas, Guerra, Amaliris, Jarocha, Danuta, Castruccio Castracani, Carlo, Papp, Tyler E., Tanaka, Naoto, Hamilton, Nolan, Triebwasser, Michael P., Ghiaccio, Valentina, Fedorky, Megan T., Gollomp, Kandace L., Bochenek, Veronica, Roche, Aoife M., Everett, John K., Cook, Emma J., Bushman, Frederic D., Teawtrakul, Nattiya, Glentis, Stavros, Kattamis, Antonis, Mui, Barbara L., Tam, Ying K., Weissman, Drew, Abdulmalik, Osheiza, Parhiz, Hamideh, Rivella, Stefano
Published in Blood (10.10.2024)
Published in Blood (10.10.2024)
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Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies
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
Published in Blood (13.11.2019)
Published in Blood (13.11.2019)
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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
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
Published in Haematologica (Roma) (27.05.2021)
Published in Haematologica (Roma) (27.05.2021)
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Journal Article
Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies
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
Published in Molecular therapy (07.04.2021)
Published in Molecular therapy (07.04.2021)
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Improved Lentiviral Vectors for the Cure of Hemoglobinopathies
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
Published in Blood (29.11.2018)
Published in Blood (29.11.2018)
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PP-14, a Novel Structurally-Enhanced Antisickling Allosteric Hemoglobin Effector, Increases Oxygen Affinity and Disrupts Hemoglobin S Polymer Formation
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
Published in Blood (13.11.2019)
Published in Blood (13.11.2019)
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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
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
Published in Haematologica (Roma) (01.05.2021)
Published in Haematologica (Roma) (01.05.2021)
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Tumor Microenvironment Remodeling by 4-Methylumbelliferone Boosts the Antitumor Effect of Combined Immunotherapy in Murine Colorectal Carcinoma
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
Published in Molecular therapy (01.09.2015)
Published in Molecular therapy (01.09.2015)
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Journal Article
2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies
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
Published in Blood (13.11.2019)
Published in Blood (13.11.2019)
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Journal Article
Adult Hemoglobin Production, Chain Rebalance, and Splice Correction in IVS2-745 Beta-Thalassemia Patient Cells Using 2'-O-Methoxyethyl Splice-Switching Oligos
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
Published in Blood (02.12.2016)
Published in Blood (02.12.2016)
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