Coordinated missplicing of TMEM14C and ABCB7 causes ring sideroblast formation in SF3B1-mutant myelodysplastic syndrome von Clough, Courtnee A., Pangallo, Joseph, Sarchi, Martina, Ilagan, Janine O., North, Khrystyna, Bergantinos, Rochelle, Stolla, Massiel C., Naru, Jasmine, Nugent, Patrick, Kim, Eunhee, Stirewalt, Derek L., Subramaniam, Arvind R., Abdel-Wahab, Omar, Abkowitz, Janis L., Bradley, Robert K., Doulatov, Sergei

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Reprogramming identifies functionally distinct stages of clonal evolution in myelodysplastic syndromes von Hsu, Jasper, Reilly, Andreea, Hayes, Brian J., Clough, Courtnee A., Konnick, Eric Q., Torok-Storb, Beverly, Gulsuner, Suleyman, Wu, David, Becker, Pamela S., Keel, Siobán B., Abkowitz, Janis L., Doulatov, Sergei

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The A946T variant of the RNA sensor IFIH1 mediates an interferon program that limits viral infection but increases the risk for autoimmunity von Gorman, Jacquelyn A, Hundhausen, Christian, Errett, John S, Stone, Amy E, Allenspach, Eric J, Ge, Yan, Arkatkar, Tanvi, Clough, Courtnee, Dai, Xuezhi, Khim, Socheath, Pestal, Kathleen, Liggitt, Denny, Cerosaletti, Karen, Stetson, Daniel B, James, Richard G, Oukka, Mohamed, Concannon, Patrick, Gale, Michael, Buckner, Jane H, Rawlings, David J

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pEVL: A Linear Plasmid for Generating mRNA IVT Templates With Extended Encoded Poly(A) Sequences von Grier, Alexandra E, Burleigh, Stephen, Sahni, Jaya, Clough, Courtnee A, Cardot, Victoire, Choe, Dongwook C, Krutein, Michelle C, Rawlings, David J, Jensen, Michael C, Scharenberg, Andrew M, Jacoby, Kyle

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The TYK2 -P1104A Autoimmune Protective Variant Limits Coordinate Signals Required to Generate Specialized T Cell Subsets von Gorman, Jacquelyn A, Hundhausen, Christian, Kinsman, Mackenzie, Arkatkar, Tanvi, Allenspach, Eric J, Clough, Courtnee, West, Samuel E, Thomas, Kerri, Eken, Ahmet, Khim, Socheath, Hale, Malika, Oukka, Mohamed, Jackson, Shaun W, Cerosaletti, Karen, Buckner, Jane H, Rawlings, David J

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Phenotypic and Genotypic Consequences of CRISPR/Cas9 Editing of the Replication Origins in the rDNA of Saccharomyces cerevisiae von Sanchez, Joseph C, Ollodart, Anja, Large, Christopher R L, Clough, Courtnee, Alvino, Gina M, Tsuchiya, Mitsuhiro, Crane, Matthew, Kwan, Elizabeth X, Kaeberlein, Matt, Dunham, Maitreya J, Raghuraman, M K, Brewer, Bonita J

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ATG4A regulates human erythroid maturation and mitochondrial clearance von Stolla, Massiel Chavez, Reilly, Andreea, Bergantinos, Rochelle, Stewart, Sintra, Thom, Neele, Clough, Courtnee A., Wellington, Rachel C., Stolitenko, Raisa, Abkowitz, Janis L., Doulatov, Sergei

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Effective, safe, and sustained correction of murine XLA using a UCOE-BTK promoter-based lentiviral vector von Seymour, Brenda J., Singh, Swati, Certo, Hannah M., Sommer, Karen, Sather, Blythe D., Khim, Socheath, Clough, Courtnee, Hale, Malika, Pangallo, Joseph, Ryu, Byoung Y., Khan, Iram F., Adair, Jennifer E., Rawlings, David J.

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Clonal Trajectories and Therapeutic Targeting of High-Risk SF3B1-Mutant Myelodysplastic Syndromes von Sarchi, Martina, Clough, Courtnee A, Gallì, Anna, Todisco, Gabriele, Creamer, J. Philip, Malcovati, Luca, Doulatov, Sergei

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Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome von Hubbard, Nicholas, Hagin, David, Sommer, Karen, Song, Yumei, Khan, Iram, Clough, Courtnee, Ochs, Hans D., Rawlings, David J., Scharenberg, Andrew M., Torgerson, Troy R.

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Modeling Clonal Progression in SF3B1-Mutant Myelodysplastic Syndrome von Sarchi, Martina, Gallì, Anna, Clough, Courtnee A, Todisco, Gabriele, Creamer, J. Philip, Malcovati, Luca, Doulatov, Sergei

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Coordinated Mis-Splicing of Multiple Mitochondrial Iron Metabolism Genes Causes Ring Sideroblast Formation in SF3B1-Mutant MDS von Clough, Courtnee, Pangallo, Joseph, Sarchi, Martina, Busch, Stephanie, Abkowitz, Janis L., Bradley, Robert K., Doulatov, Sergei

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Probing Aberrant Splicing in a Novel Model of SF3B1-Mutant Myelodysplastic Syndromes von Clough, Courtnee, Busch, Stephanie, Pangallo, Joey, Bradley, Robert K., Doulatov, Sergei

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ATG4A is a Novel Regulator of Mitophagy during Human Erythropoiesis von Stolla, Massiel Chavez, Thom, Neele, Reilly, Andreea, Clough, Courtnee, Abkowitz, Janis L., Doulatov, Sergei

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Mitotic Dysregulation Sensitizes Malignant Stem Cells to CHK1 Inhibition in SF3B1-Mutant Myeloid Neoplasms von Sarchi, Martina, Clough, Courtnee A., Crosse, Edie I., Kim, Jason, Baquero Galvis, Laura D., Gallì, Anna, Creamer, J. Philip, Stewart, Sintra, Pozzi, Sara, Molteni, Elisabetta, Elena, Chiara, Bradley, Robert K., Malcovati, Luca, Doulatov, Sergei

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A Shared SF3B1 Neoantigen Is Presented on Primary Malignant Cells and Induced Pluripotent Stem Cell-Derived Hematopoietic Lines von Biernacki, Melinda Ann, Foster, Kimberly A, Clough, Courtnee, Busch, Stephanie, Cummings, Carrie, Oehler, Vivian G., Stirewalt, Derek, Doulatov, Sergei, Bleakley, Marie

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3009 – MIS-SPLICING OF TMEM14C ALTERS PORPHYRIN AVAILABILITY PROMOTING RING SIDEROBLAST FORMATION IN SF3B1-MUTANT MYELODYSPLASTIC SYNDROMES von Clough, Courtnee, Pangallo, Joseph, Sarchi, Martina, Ilagan, Janine, North, Khrystyna, Bergantinos, Rochelle, Naru, Jasmine, Nugent, Patrick, Kim, Eunhee, Stirewalt, Derek, Subramaniam, Arvind, Abdel-Wahab, Omar, Abkowitz, Janis, Bradley, Robert, Doulatov, Sergei

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569. Precision Editing of the WAS Locus via Homologous Recombination in Primary Human Hematopoietic Cells Mediated by Either TALEN or CRISPR/Cas Nucleases von Khan, Iram F., Wang, Yupeng, Clough, Courtnee, Singh, Swati, Hung, King, Rawlings, David J.

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132. Targeting the BTK Locus in Primary Human Hematopoietic Cells with TALENs and AAV Donor Template von Clough, Courtnee, Wang, Yupeng, Khan, Iram F., Singh, Swati, Hung, King, Rawlings, David J.

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334. Improved MegaTAL Endonuclease Engineering Strategy for Gene Modification Applications von Wang, Yupeng, Khan, Iram F., Clough, Courtnee, Scharenberg, Andrew M., Rawlings, David J.

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