Challenges and promises for the development of donor-independent platelet transfusions von Lambert, Michele P., Sullivan, Spencer K., Fuentes, Rudy, French, Deborah L., Poncz, Mortimer

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Comparative analysis of human ex vivo–generated platelets vs megakaryocyte-generated platelets in mice: a cautionary tale von Wang, Yuhuan, Hayes, Vincent, Jarocha, Danuta, Sim, Xiuli, Harper, Dawn C., Fuentes, Rudy, Sullivan, Spencer K., Gadue, Paul, Chou, Stella T., Torok-Storb, Beverly J., Marks, Michael S., French, Deborah L., Poncz, Mortimer

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Clonal genetic and hematopoietic heterogeneity among human-induced pluripotent stem cell lines von Mills, Jason A., Wang, Kai, Paluru, Prasuna, Ying, Lei, Lu, Lin, Galvão, Aline M., Xu, Dongbin, Yao, Yu, Sullivan, Spencer K., Sullivan, Lisa M., Mac, Helen, Omari, Amel, Jean, Jyh-Chang, Shen, Steve, Gower, Adam, Spira, Avi, Mostoslavsky, Gustavo, Kotton, Darrell N., French, Deborah L., Weiss, Mitchell J., Gadue, Paul

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Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A von George, Lindsey A, Monahan, Paul E, Eyster, M. Elaine, Sullivan, Spencer K, Ragni, Margaret V, Croteau, Stacy E, Rasko, John E.J, Recht, Michael, Samelson-Jones, Benjamin J, MacDougall, Amy, Jaworski, Kristen, Noble, Robert, Curran, Marla, Kuranda, Klaudia, Mingozzi, Federico, Chang, Tiffany, Reape, Kathleen Z, Anguela, Xavier M, High, Katherine A

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The negative impact of Wnt signaling on megakaryocyte and primitive erythroid progenitors derived from human embryonic stem cells von Paluru, Prasuna, Hudock, Kristin M., Cheng, Xin, Mills, Jason A., Ying, Lei, Galvão, Aline M., Lu, Lin, Tiyaboonchai, Amita, Sim, Xiuli, Sullivan, Spencer K., French, Deborah L., Gadue, Paul

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Novel C16orf57 mutations in patients with Poikiloderma with Neutropenia: bioinformatic analysis of the protein and predicted effects of all reported mutations von Colombo, Elisa A, Bazan, J Fernando, Negri, Gloria, Gervasini, Cristina, Elcioglu, Nursel H, Yucelten, Deniz, Altunay, Ilknur, Cetincelik, Umram, Teti, Anna, Del Fattore, Andrea, Luciani, Matteo, Sullivan, Spencer K, Yan, Albert C, Volpi, Ludovica, Larizza, Lidia

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FLI1 level during megakaryopoiesis affects thrombopoiesis and platelet biology von Vo, Karen K., Jarocha, Danuta J., Lyde, Randolph B., Hayes, Vincent, Thom, Christopher S., Sullivan, Spencer K., French, Deborah L., Poncz, Mortimer

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Efficient iPS cell generation from blood using episomes and HDAC inhibitors von Hubbard, Jesse J, Sullivan, Spencer K, Mills, Jason A, Hayes, Brian J, Torok-Storb, Beverly J, Ramakrishnan, Aravind

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Generation of poikiloderma with neutropenia (PN) induced pluripotent stem cells (iPSCs) von Mills, Jason A., Hudock, Kristin M., Sullivan, Spencer K., Herrera, Pamela, Sullivan, Lisa M., Gadue, Paul, French, Deborah L.

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Patient-derived induced pluripotent stem cells recapitulate hematopoietic abnormalities of juvenile myelomonocytic leukemia von Gandre-Babbe, Shilpa, Paluru, Prasuna, Aribeana, Chiaka, Chou, Stella T., Bresolin, Silvia, Lu, Lin, Sullivan, Spencer K., Tasian, Sarah K., Weng, Julie, Favre, Helene, Choi, John K., French, Deborah L., Loh, Mignon L., Weiss, Mitchell J.

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High-level transgene expression in induced pluripotent stem cell–derived megakaryocytes: correction of Glanzmann thrombasthenia von Sullivan, Spencer K., Mills, Jason A., Koukouritaki, Sevasti B., Vo, Karen K., Lyde, Randolph B., Paluru, Prasuna, Zhao, Guoha, Zhai, Li, Sullivan, Lisa M., Wang, Yuhuan, Kishore, Siddharth, Gharaibeh, Eyad Z., Lambert, Michele P., Wilcox, David A., French, Deborah L., Poncz, Mortimer, Gadue, Paul

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A(nother) day in the life of neonatal platelets von Sullivan, Spencer K., Poncz, Mortimer

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Inducible Gata1 suppression expands megakaryocyte-erythroid progenitors from embryonic stem cells von Noh, Ji-Yoon, Gandre-Babbe, Shilpa, Wang, Yuhuan, Hayes, Vincent, Yao, Yu, Gadue, Paul, Sullivan, Spencer K, Chou, Stella T, Machlus, Kellie R, Italiano, Jr, Joseph E, Kyba, Michael, Finkelstein, David, Ulirsch, Jacob C, Sankaran, Vijay G, French, Deborah L, Poncz, Mortimer, Weiss, Mitchell J

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Infused factor VIII–expressing platelets or megakaryocytes as a novel therapeutic strategy for hemophilia A von Lyde, Randolph B., Ahn, Hyun Sook, Vo, Karen K., Jarocha, Danuta J., Tkaczynski, John, Treffeisen, Elsa, Sullivan, Spencer K., Camire, Rodney M., Sabatino, Denise E., French, Deborah L., Poncz, Mortimer

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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant von George, Lindsey A, Sullivan, Spencer K, Giermasz, Adam, Rasko, John E.J, Samelson-Jones, Benjamin J, Ducore, Jonathan, Cuker, Adam, Sullivan, Lisa M, Majumdar, Suvankar, Teitel, Jerome, McGuinn, Catherine E, Ragni, Margaret V, Luk, Alvin Y, Hui, Daniel, Wright, J. Fraser, Chen, Yifeng, Liu, Yun, Wachtel, Katie, Winters, Angela, Tiefenbacher, Stefan, Arruda, Valder R, van der Loo, Johannes C.M, Zelenaia, Olga, Takefman, Daniel, Carr, Marcus E, Couto, Linda B, Anguela, Xavier M, High, Katherine A

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Inducible Gata1 suppression expands megakaryocyte-erythroid progenitors from embryonic stem cells von Noh, Ji-Yoon, Gandre-Babbe, Shilpa, Wang, Yuhuan, Hayes, Vincent, Yao, Yu, Gadue, Paul, Sullivan, Spencer K., Chou, Stella T., Machlus, Kellie R., Italiano, Joseph E., Kyba, Michael, Finkelstein, David, Ulirsch, Jacob C., Sankaran, Vijay G., French, Deborah L., Poncz, Mortimer, Weiss, Mitchell J.

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Health-Related Quality of Life in Adults with Hemophilia B after Receiving Gene Therapy with Fidanacogene Elaparvovec von von Mackensen, Sylvia, Ducore, Jonathan M., George, Lindsey A., Giermasz, Adam, McGuinn, Catherine, Rasko, John E. J., Samelson-Jones, Ben J, Sullivan, Spencer K., Teitel, Jerome M., Chhabra, Amit, Fang, Annie F., O'Brien, Amanda, Plonski, Frank, Rupon, Jeremy, Smith, Lynne, Winburn, Ian

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Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy von Samelson-Jones, Ben J., Sullivan, Spencer K., Rasko, John E.J., Giermasz, Adam, George, Lindsey A., Ducore, Jonathan M., Teitel, Jerome M., McGuinn, Catherine E., O'Brien, Amanda, Winburn, Ian, Smith, Lynne M, Chhabra, Amit, Rupon, Jeremy

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Fli1 Expression Level Affects Megakaryopoiesis and Thrombopoiesis: More Is Not Always a Bad Thing von Vo, Karen K, Jarocha, Danuta Jadwiga, Lyde, Randolph B, Sullivan, Spencer K., French, Deborah, Poncz, Mortimer

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Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year von George, Lindsey A., Sullivan, Spencer K., Rasko, John E.J., Giermasz, Adam, Samelson-Jones, Benjamin J., Ducore, Jonathan M., Teitel, Jerome M., McGuinn, Catherine E., Runowski, Alexa R., Wright, Fraser, Anguela, Xavier M., High, Katherine A., Rybin, Denis, Murphy, John E., Rupon, Jeremy

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