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SaveShare Save Share Save Outcomes of Immunosuppressive Therapy in Children Less Than Three Years of Age with Idiopathic Severe Aplastic Anemia: A North American Collaboration
Dayagi, Talya Wittmann; Willis, Laura; Corriveau-Bourque, Catherine; Cada, Michaela; Rogers, Zora R.; Nakano, Taizo A.; Olson, Timothy S.; Wang, Winfred C.; Gillio, Alfred; Coates, Thomas D.; Chawla, Anjulika; Castillo, Paul; Kurre, Peter; Gamper, Christopher; Arnold, Staci D.; Joshi, Sarita; Geddis, Amy E.; Boklan, Jessica; Rothman, Jennifer A.; Huang, James N.; Kupfer, Gary M.; Glader, Bertil; Walkovich, Kelly J.; Thompson, Alexis; Hanna, Rabi; Williams, David A.; Shimamura, Akiko; Dror, Yigal Share Save Hematologic Cancer after Gene Therapy for Cerebral AdrenoleukodystrophyDuncan, Christine N.; Bledsoe, Jacob R.; Grzywacz, Bartosz; Beckman, Amy; Bonner, Melissa; Eichler, Florian S.; Kuhl, Joern-Sven; Harris, Marian H.; Slauson, Sarah; Colvin, Richard A.; Prasad, Vinod K.; Downey, Gerald F.; Pierciey, Francis J.; Kinney, Melissa A.; Foos, Marianna; Lodaya, Ankit; Floro, Nicole; Parsons, Geoffrey; Dietz, Andrew C.; Gupta, Ashish O.; Orchard, Paul J.; Thakar, Himal L.; Williams, David A. Share Save Lentiviral Gene Therapy for Cerebral AdrenoleukodystrophyEichler, Florian; Duncan, Christine N.; Musolino, Patricia L.; Lund, Troy C.; Gupta, Ashish O.; De Oliveira, Satiro; Thrasher, Adrian J.; Aubourg, Patrick; Kuehl, Joern-Sven; Loes, Daniel J.; Amartino, Hernan; Smith, Nicholas; Fernandes, Juliana Folloni; Sevin, Caroline; Sankar, Raman; Hussain, Shaun A.; Gissen, Paul; Dalle, Jean-Hugues; Platzbecker, Uwe; Downey, Gerald F.; McNeil, Elizabeth; Demopoulos, Laura; Dietz, Andrew C.; Thakar, Himal L.; Orchard, Paul J.; Williams, David A. Share Save Clonal selection of hematopoietic stem cells after gene therapy for sickle cell diseaseChapman, Michael Spencer; Cull, Alyssa H.; Ciuculescu, Marioara F.; Esrick, Erica B.; Mitchell, Emily; Jung, Hyunchul; O'Neill, Laura; Roberts, Kirsty; Fabre, Margarete A.; Williams, Nicholas; Nangalia, Jyoti; Quinton, Joanne; Fox, James M.; Pellin, Danilo; Makani, Julie; Armant, Myriam; Williams, David A.; Campbell, Peter J.; Kent, David G. Share Save Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophyLauer, Arne; Speroni, Samantha L.; Choi, Myoung; Da, Xiao; Duncan, Christine; McCarthy, Siobhan; Krishnan, Vijai; Lusk, Cole A.; Rohde, David; Hansen, Mikkel Bo; Kalpathy-Cramer, Jayashree; Loes, Daniel J.; Caruso, Paul A.; Williams, David A.; Mouridsen, Kim; Emblem, Kyrre E.; Eichler, Florian S.; Musolino, Patricia L. Share Save Rac2, a hematopoiesis-specific Rho GTPase, specifically regulates mast cell protease gene expression in bone marrow-derived mast cellsGu, Y; Byrne, MC; Paranavitana, NC; Aronow, B; Siefring, JE; D'Souza, M; Horton, HF; Quilliam, LA; Williams, DA Share Save Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trialsCornetta, Kenneth; Yao, Jing; House, Kimberley; Duffy, Lisa; Adusumilli, Prasad S.; Beyer, Rachel; Booth, Claire; Brenner, Malcolm; Curran, Kevin; Grilley, Bambi; Heslop, Helen; Hinrichs, Christian S.; Kaplan, Rosandra N.; Kiem, Hans-Peter; Kochenderfer, James; Kohn, Donald B.; Mailankody, Sham; Norberg, Scott M.; O'Cearbhaill, Roisin E.; Pappas, Jennifer; Park, Jae; Ramos, Carlos; Ribas, Antonio; Riviere, Isabelle; Rosenberg, Steven A.; Sauter, Craig; Shah, Nirali N.; Slovin, Susan F.; Thrasher, Adrian; Williams, David A.; Lin, Tsai-Yu Share Save Share Save Development of a double shmiR lentivirus effectively targeting both BCL11A and ZNF410 for enhanced induction of fetal hemoglobin to treat ll-hemoglobinopathies brLiu, Boya; Brendel, Christian; Vinjamur, Divya S.; Zhou, Yu; Harris, Chad; McGuinness, Meaghan; Manis, John P.; Bauer, Daniel E.; Xu, Haiming; Williams, David A. Share Save Validation of a small molecule inhibitor of PDE6D-RAS interaction with favorable anti-leukemic effectsNunes, Sara Canovas; De Vita, Serena; Anighoro, Andrew; Autelitano, Francois; Beaumont, Edward; Klingbeil, Pamela; McGuinness, Meaghan; Duvert, Beatrice; Harris, Chad; Yang, Lu; Pokharel, Sheela Pangeni; Chen, Chun-Wei; Ermann, Monika; Williams, David A.; Xu, Haiming Share Save Share Save Clonal hematopoiesis in sickle cell diseaseLiggett, L. Alexander; Cato, Liam D.; Weinstock, Joshua S.; Zhang, Yingze; Nouraie, S. Mehdi; Gladwin, Mark T.; Garrett, Melanie E.; Ashley-Koch, Allison; Telen, Marilyn J.; Custer, Brian; Kelly, Shannon; Dinardo, Carla L.; Sabino, Ester C.; Loureiro, Paula; Carneiro-Proietti, Anna B.; Maximo, Claudia; Reiner, Alexander P.; Abecasis, Goncalo R.; Williams, David A.; Natarajan, Pradeep; Bick, Alexander G.; Sankaran, Vijay G. Share Save Congenital X-linked neutropenia with myelodysplasia and somatic tetraploidy due to a germline mutation in SEPT6Renella, Raffaele; Gagne, Katelyn; Beauchamp, Ellen; Fogel, Jonathan; Perlov, Aleksej; Sola, Mireia; Schlaeger, Thorsten; Hofmann, Inga; Shimamura, Akiko; Ebert, Benjamin L.; Schmitz-Abe, Klaus; Markianos, Kyriacos; Murphy, Kristi; Sun, Liang; Rockowitz, Shira; Sliz, Piotr; Campagna, Dean R.; Springer, Timothy A.; Bahl, Christopher; Agarwal, Suneet; Fleming, Mark D.; Williams, David A. Share Save Share Save Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell DiseaseEsrick, Erica B.; Lehmann, Leslie E.; Biffi, Alessandra; Achebe, Maureen; Brendel, Christian; Ciuculescu, Marioara F.; Daley, Heather; MacKinnon, Brenda; Morris, Emily; Federico, Amy; Abriss, Daniela; Boardman, Kari; Khelladi, Radia; Shaw, Kit; Negre, Helene; Negre, Olivier; Nikiforow, Sarah; Ritz, Jerome; Pai, Sung-Yun; London, Wendy B.; Dansereau, Colleen; Heeney, Matthew M.; Armant, Myriam; Manis, John P.; Williams, David A. Share Save HACE1 Prevents Lung Carcinogenesis via Inhibition of RAC-Family GTPasesKogler, Melanie; Tortola, Luigi; Negri, Gian Luca; Leopoldi, Alexandra; El-Naggar, Amal M.; Mereiter, Stefan; Gomez-Diaz, Carlos; Nitsch, Roberto; Tortora, Davide; Kavirayani, Anoop M.; Gapp, Bianca, V; Rao, Shuan; Uribesalgo, Iris; Hoffmann, David; Cikes, Domagoj; Novatchkova, Maria; Williams, David A.; Trent, Jeffrey M.; Ikeda, Fumiyo; Daugaard, Mads; Hagelkruys, Astrid; Sorensen, Poul H.; Penninger, Josef M. Share Save Share Save 
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