KBTBD4-mediated reduction of MYC is critical for hematopoietic stem cell expansion upon UM171 treatment

J Chagraoui, S Girard, L Mallinger, N Mayotte… - Blood, 2024 - ashpublications.org
Ex vivo expansion of hematopoietic stem cells (HSCs) is gaining importance for cell and
gene therapy, and requires a shift from dormancy state to activation and cycling. However …

UM171 enhances fitness and engraftment of gene modified hematopoietic stem cells from sickle cells disease patients

B Liu, D Klatt, Y Zhou, JP Manis, G Sauvageau… - Blood Advances, 2024 - Elsevier
Hematopoietic stem cell transplantation with lentiviral vector (LVV) transduced autologous
cells has proven an effective therapeutic strategy for sickle cell disease (SCD). However, ex …

Structural mimicry of UM171 and neomorphic cancer mutants co-opts E3 ligase KBTBD4 for HDAC1/2 recruitment

Z Chen, G Chi, T Balo, X Chen, B Montes, SC Clifford… - bioRxiv, 2024 - biorxiv.org
Neomorphic mutations and drugs can elicit unanticipated effects that hinder mechanistic
understanding for clinical practice. Recurrent indel mutations in the Kelch domain of the …

[HTML][HTML] Ex vivo hematopoietic stem cell expansion technologies: recent progress, applications, and open questions

GA Meaker, AC Wilkinson - Experimental Hematology, 2023 - Elsevier
Hematopoietic stem cells (HSCs) are a rare but potent cell type that support life-long
hematopoiesis and will stably regenerate the entire blood and immune system following …