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Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations Tricoli, Lucas; Sase, Sunetra; Hacker, Julia L.; Pham, Vi; Chappell, Maxwell; Breda, Laura; Hurwitz, Stephanie N.; Tanaka, Naoto; Castracani, Carlo Castruccio; Guerra, Amaliris; Hou, Zhongqi; Schlotawa, Lars; Radhakrishnan, Karthikeyan; Hogenauer, Matthew; Roche, Aoife; Everett, John; Bushman, Frederic; Kurre, Peter; Ahrens-Nicklas, Rebecca; Adang, Laura A.; Vanderver, Adeline L.; Rivella, Stefano Share Save
Restoring hematopoietic stem and progenitor cell function in Fancc-/- mice by in situ delivery of RNA lipid nanoparticles Banda, Omar; Adams, Sarah E.; Omer, Linah; Jung, Seul K.; Said, Hooda; Phoka, Theerapat; Tam, Ying; Weissman, Drew; Rivella, Stefano; Alameh, Mohamad-Gabriel; Kurre, Peter Share Save
Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency Pham, Vi; Tricoli, Lucas; Hong, Xinying; Wongkittichote, Parith; Castracani, Carlo Castruccio; Guerra, Amaliris; Schlotawa, Lars; Adang, Laura A.; Kuhs, Amanda; Cassidy, Margaret M.; Kane, Owen; Tsai, Emily; Presa, Maximiliano; Lutz, Cathleen; Rivella, Stefano B.; Ahrens-Nicklas, Rebecca C. Share Save
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Elevated CDKN1A (P21) mediates β-thalassemia erythroid apoptosis, but its loss does not improve β-thalassemic erythropoiesis Liang, Raymond; Lin, Miao; Menon, Vijay; Qiu, Jiajing; Menon, Anagha; Breda, Laura; Arif, Tasleem; Rivella, Stefano; Ghaffari, Saghi Share Save
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Transferrin receptor 2 (Tfr2) genetic deletion makes transfusion-independent a murine model of transfusion-dependent β-thalassemia Di Modica, Simona Maria; Tanzi, Emanuele; Olivari, Violante; Lidonnici, Maria Rosa; Pettinato, Mariateresa; Pagani, Alessia; Tiboni, Francesca; Furiosi, Valeria; Silvestri, Laura; Ferrari, Giuliana; Rivella, Stefano; Nai, Antonella Share Save
DNA binding to TLR9 expressed by red blood cells promotes innate immune activation and anemia Lam, L. K. Metthew; Murphy, Sophia; Kokkinaki, Dimitra; Venosa, Alessandro; Sherrill-Mix, Scott; Casu, Carla; Rivella, Stefano; Weiner, Aaron; Park, Jeongho; Shin, Sunny; Vaughan, Andrew E.; Hahn, Beatrice H.; John, Audrey R. Odom; Meyer, Nuala J.; Hunter, Christopher A.; Worthen, G. Scott; Mangalmurti, Nilam S. Share Save
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The EHA Research Roadmap: Anemias Iolascon, Achille; Rivella, Stefano; Anagnou, Nicholas P.; Camaschella, Clara; Swinkels, Dorine; Muckenthaler, Martina U.; Porto, Graca; Barcellini, Wilma; Andolfo, Immacolata; Risitano, Antonio M.; Kattamis, Antonis; Cappellini, Maria Domenica; Taher, Ali T.; De Franceschi, Lucia; Rees, David; Russo, Roberta; Tamary, Hannah; Stauder, Reinhard; Girelli, Domenico Share Save
Inclusion of a short hairpin RNA targeting BCL11A into a β-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 Share Save
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