The A946T variant of the RNA sensor IFIH1 mediates an interferon program that limits viral infection but increases the risk for autoimmunity JA Gorman, C Hundhausen, JS Errett, AE Stone, EJ Allenspach, Y Ge, ... Nature immunology 18 (7), 744-752, 2017 | 141 | 2017 |
Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome N Hubbard, D Hagin, K Sommer, Y Song, I Khan, C Clough, HD Ochs, ... Blood, The Journal of the American Society of Hematology 127 (21), 2513-2522, 2016 | 139 | 2016 |
pEVL: a linear plasmid for generating mRNA IVT templates with extended encoded poly (A) sequences AE Grier, S Burleigh, J Sahni, CA Clough, V Cardot, DC Choe, MC Krutein, ... Molecular Therapy-Nucleic Acids 5, 2016 | 91 | 2016 |
Coordinated missplicing of TMEM14C and ABCB7 causes ring sideroblast formation in SF3B1-mutant myelodysplastic syndrome CA Clough, J Pangallo, M Sarchi, JO Ilagan, K North, R Bergantinos, ... Blood, The Journal of the American Society of Hematology 139 (13), 2038-2049, 2022 | 69 | 2022 |
Reprogramming identifies functionally distinct stages of clonal evolution in myelodysplastic syndromes J Hsu, A Reilly, BJ Hayes, CA Clough, EQ Konnick, B Torok-Storb, ... Blood, The Journal of the American Society of Hematology 134 (2), 186-198, 2019 | 60 | 2019 |
The TYK2-P1104A Autoimmune Protective Variant Limits Coordinate Signals Required to Generate Specialized T Cell Subsets JA Gorman, C Hundhausen, M Kinsman, T Arkatkar, EJ Allenspach, ... Frontiers in immunology 10, 44, 2019 | 43 | 2019 |
Targeted Gene Editing Restores Regulated CD40L Expression and Function in X-HIGM T cells. NW Hubbard, D Hagin, K Sommer, Y Song, I Khan, C Clough, HD Ochs, ... The Journal of Immunology 196 (1_Supplement), 214.28-214.28, 2016 | 19 | 2016 |
Phenotypic and Genotypic Consequences of CRISPR/Cas9 Editing of the Replication Origins in the rDNA of Saccharomyces cerevisiae JC Sanchez, A Ollodart, CRL Large, C Clough, GM Alvino, M Tsuchiya, ... Genetics 213 (1), 229-249, 2019 | 12 | 2019 |
Effective, safe, and sustained correction of murine XLA using a UCOE-BTK promoter-based lentiviral vector BJ Seymour, S Singh, HM Certo, K Sommer, BD Sather, S Khim, ... Molecular Therapy-Methods & Clinical Development 20, 635-651, 2021 | 11 | 2021 |
132. Targeting the BTK locus in primary human hematopoietic cells with TALENs and AAV donor template C Clough, Y Wang, IF Khan, S Singh, K Hung, DJ Rawlings Molecular Therapy 24, S54, 2016 | 7 | 2016 |
ATG4A regulates human erythroid maturation and mitochondrial clearance MC Stolla, A Reilly, R Bergantinos, S Stewart, N Thom, CA Clough, ... Blood Advances 6 (12), 3579-3589, 2022 | 6 | 2022 |
The TYK2-P1104A Autoimmune Protective Variant Limits Coordinate Signals Required to Generate Specialized T Cell Subsets. Front Immunol. 2019; 10: 44 JA Gorman, C Hundhausen, M Kinsman, T Arkatkar, EJ Allenspach, ... | 6 | 2019 |
238. BTK-Promoter LV Vectors Utilizing Conserved Intron Element Mediate Functional Rescue in Murine XLA S Singh, B Seymour, K Sommer, I Khan, S Khim, M Hale, C Clough, ... Molecular Therapy 23, S93, 2015 | 4 | 2015 |
Mis-splicing of mitotic regulators sensitizes SF3B1-mutated human HSCs to CHK1 inhibition M Sarchi, CA Clough, EI Crosse, J Kim, LD Baquero Galvis, N Aydinyan, ... Blood Cancer Discovery, 2024 | 3 | 2024 |
Coordinated mis-splicing of multiple mitochondrial iron metabolism genes causes ring sideroblast formation in SF3B1-mutant MDS C Clough, J Pangallo, M Sarchi, S Busch, JL Abkowitz, RK Bradley, ... Blood 136, 4, 2020 | 2 | 2020 |
A Shared SF3B1 Neoantigen Is Presented on Primary Malignant Cells and Induced Pluripotent Stem Cell-Derived Hematopoietic Lines MA Biernacki, KA Foster, C Clough, S Busch, C Cummings, VG Oehler, ... Blood 136, 13-14, 2020 | 2 | 2020 |
Clonal Trajectories and Therapeutic Targeting of High-Risk SF3B1-Mutant Myelodysplastic Syndromes M Sarchi, CA Clough, A Gallì, G Todisco, JP Creamer, L Malcovati, ... Blood 140 (Supplement 1), 211-212, 2022 | 1 | 2022 |
Modeling Clonal Progression in SF3B1-Mutant Myelodysplastic Syndrome M Sarchi, A Gallì, CA Clough, G Todisco, JP Creamer, L Malcovati, ... Blood 138, 149, 2021 | 1 | 2021 |
ATG4A is a Novel Regulator of Mitophagy during Human Erythropoiesis MC Stolla, N Thom, A Reilly, C Clough, JL Abkowitz, S Doulatov Blood 134, 3522, 2019 | 1 | 2019 |
569. Precision Editing of the WAS Locus via Homologous Recombination in Primary Human Hematopoietic Cells Mediated by Either TALEN or CRISPR/Cas Nucleases IF Khan, Y Wang, C Clough, S Singh, K Hung, DJ Rawlings Molecular Therapy 24, S227, 2016 | 1 | 2016 |