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Nature|January 24, 2020
Selective inhibition of the BD2 bromodomain of BET proteins in prostate cancerEmily J Faivre, Keith F McDaniel, Daniel H Albert, et al.
Nature Genetics|August 18, 2014
JAGN1 deficiency causes aberrant myeloid cell homeostasis and congenital neutropeniaKaan Boztug, Päivi M Järvinen, Elisabeth Salzer, et al.
Journal of Medicinal Chemistry|April 4, 2017
Fragment-Based, Structure-Enabled Discovery of Novel Pyridones and Pyridone Macrocycles as Potent Bromodomain and Extra-Terminal Domain (BET) Family Bromodomain InhibitorsLe Wang, John K Pratt, Todd Soltwedel, et al.
Bone Marrow Transplantation|March 25, 2022
Allogeneic hematopoietic stem cell transplantation for adult HLH: a retrospective study by the chronic malignancies and inborn errors working parties of EBMTRafal Machowicz, Felipe Suarez, Wieslaw Wiktor-Jedrzejczak, et al.
Science Translational Medicine|March 20, 2015
Exploiting selective BCL-2 family inhibitors to dissect cell survival dependencies and define improved strategies for cancer therapyJoel D Leverson, Darren C Phillips, Michael J Mitten, et al.
Blood Advances|June 20, 2025
Allogeneic hematopoietic stem cell transplantation for STAT3 hyper-IgE syndrome: a worldwide studyChristo Tsilifis, Johannes Raedler, Joanna Renke, et al.
Blood Advances|March 3, 2026
Outcome of Second Conditioned Allogeneic Stem Cell Transplant in Children with Non-SCID Inborn Errors of ImmunityZohreh Nademi, Su-Han Lum, Patrick Gilbert, et al.
Blood|January 31, 2022
Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysisMichael H Albert, Mary A Slatter, Andrew R Gennery, et al.
Blood|October 24, 2022
Hypomorphic RAG deficiency: impact of disease burden on survival and thymic recovery argues for early diagnosis and HSCTC Schuetz, J Gerke, M Ege, et al.
The Lancet. Haematology|April 15, 2019
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical studyFrancesca Ferrua, Maria Pia Cicalese, Stefania Galimberti, et al.
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