Highlighted Publications

Mapping the cellular origin and early evolution of leukemia in Down syndrome

Science, Jul 09, 2021

We utilized CRISPR/Cas9-mediated genome editing in human primary disomic and trisomic hematopoietic stem cells to generate an in vivo model of Down syndrome associated leukemia evolution. This humanized model enabled us to study the mechanism of predisposition, preleukemia initiation and leukemic transformation in order to identify potential therapeutic targets of the disease.

Functional profiling of single CRISPR/Cas9-edited human long-term hematopoietic stem cells

Nat Communications, Oct 18, 2019

We established a state-of-the-art electroporation-based approach of CRISPR/Cas9-mediated genome editing for human primary long-term hematopoietic stem cells. Our methodology allows rapid interrogation of gene deletions, splice variants and fusion genes in order to study gene function relationships in normal and malignant blood stem cells.

Publications Archive

Epitope base editing: Non-malignant stem cells are going undercover

Cell Stem Cell, Nov 13, 2023

Saniei S, Wagenblast E

Down syndrome and leukemia: from basic mechanisms to clinical advances

Haematologica, Oct 02, 2023

Baruchel A, Bourquin JP, Crispino J, Cuartero S, Hasle H, Hitzler J, Klusmann JH, Izraeli S, Lane AA, Malinge S, Rabin KR, Roberts I, Ryeom S, Tasian SK, Wagenblast E

A primary hierarchically organized patient-derived model enables in depth interrogation of stemness driven by the coding and non-coding genome

Leukemia, Sep 21, 2022

Boutzen H, Madani Tonekaboni SA, Chan-Seng-Yue M, Murison A, Takayama N, Mbong N, Wagenblast E, Orouji E, Arruda A, Mitchell A, Notta F, Minden MD, Lupien M, Kaufmann KB, Dick JE

Identification of the global miR-130a targetome reveals a role for TBL1XR1 in hematopoietic stem cell self-renewal and t(8;21) AML

Cell Rep, Mar 08, 2022

Krivdova G, Voisin V, Schoof EM, Marhon SA, Murison A, McLeod JL, Gabra MM, Zeng AGX, Aigner S, Yee BA, Shishkin AA, Van Nostrand EL, Hermans KG, Trotman-Grant AC, Mbong N, Kennedy JA, Gan OI, Wagenblast E, De Carvalho DD, Salmena L, Minden MD, Bader GD, Yeo GW, Dick JE, Lechman ER

TFEB-mediated endolysosomal activity controls human hematopoietic stem cell fate

Cell Stem Cell, Aug 02, 2021

García-Prat L, Kaufmann KB, Schneiter F, Voisin V, Murison A, Chen J, Chan-Seng-Yue M, Gan OI, McLeod JL, Smith SA, Shoong MC, Parris D, Pan K, Zeng AGX, Krivdova G, Gupta K, Takayanagi SI, Wagenblast E, Wang W, Lupien M, Schroeder T, Xie SZ, Dick JE

Mapping the cellular origin and early evolution of leukemia in Down syndrome

Science, Jul 09, 2021

Wagenblast E, Araújo J, Gan OI, Cutting SK, Murison A, Krivdova G, Azkanaz M, McLeod JL, Smith SA, Gratton BA, Marhon SA, Gabra M, Medeiros JJF, Manteghi S, Chen J, Chan-Seng-Yue M, Garcia-Prat L, Salmena L, De Carvalho DD, Abelson S, Abdelhaleem M, Chong K, Roifman M, Shannon P, Wang JCY, Hitzler JK, Chitayat D, Dick JE, Lechman ER

Sphingosine-1-phosphate receptor 3 potentiates inflammatory programs in normal and leukemia stem cells to promote differentiation

Blood Cancer Discov, Jan 01, 2021

Xie SZ, Kaufmann KB, Wang W, Chan-Seng-Yue M, Gan OI, Laurenti E, Garcia-Prat L, Takayanagi SI, Ng SWK, Xu C, Zeng AGX, Jin L, McLeod J, Wagenblast E, Mitchell A, Kennedy JA, Liu Q, Boutzen H, Kleinau M, Jargstorf J, Holmes G, Zhang Y, Voisin V, Bader GD, Wang JCY, Hannun YA, Luberto C, Schroeder T, Minden MD, Dick JE

Characterization of universal features of partially methylated domains across tissues and species

Epigenetics Chromatin, Oct 02, 2020

Decato BE, Qu J, Ji X, Wagenblast E, Knott SRV, Hannon GJ, Smith AD

Sphingolipid Modulation Activates Proteostasis Programs to Govern Human Hematopoietic Stem Cell Self-Renewal

Cell Stem Cell, Nov 07, 2019

Xie SZ, Garcia-Prat L, Voisin V, Ferrari R, Gan OI, Wagenblast E, Kaufmann KB, Zeng AGX, Takayanagi SI, Patel I, Lee EK, Jargstorf J, Holmes G, Romm G, Pan K, Shoong M, Vedi A, Luberto C, Minden MD, Bader GD, Laurenti E, Dick JE

Functional profiling of single CRISPR/Cas9-edited human long-term hematopoietic stem cells

Nat Communications, Oct 18, 2019

Wagenblast E, Azkanaz M, Smith SA, Shakib L, McLeod JL, Krivdova G, Araújo J, Shultz LD, Gan OI, Dick JE, Lechman ER