Guy Sauvageau
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View article: High IL1R1 expression predicts poor survival and benefit from stem cell transplant in intermediate-risk acute myeloid leukemia from the Leucegene cohort
High IL1R1 expression predicts poor survival and benefit from stem cell transplant in intermediate-risk acute myeloid leukemia from the Leucegene cohort Open
Background There is an unmet clinical need to identify patients with acute myeloid leukemia and intermediate-risk cytogenetics who benefit from allogeneic hematopoietic stem cell transplantation in first remission, especially among those w…
View article: <i>TP53</i>-mutant AML with ribosomal gene loss exhibits impaired protein translation and sensitivity to HSP90 inhibition
<i>TP53</i>-mutant AML with ribosomal gene loss exhibits impaired protein translation and sensitivity to HSP90 inhibition Open
TP53 -mutated acute myeloid leukemia (AML) represents a particularly aggressive and therapeutically refractory subtype of the disease. While recurrent chromosomal abnormalities such as -5/del(5q), -7/del(7q), and del(17p) are well studied …
View article: Predictive Gene Discovery with EPCY: A Density-Based Alternative to DE analysis
Predictive Gene Discovery with EPCY: A Density-Based Alternative to DE analysis Open
Identifying predictive genes from high-throughput data remains a key challenge in biomedical research. Most current approaches rely on statistical tests to select differentially expressed genes (DEGs), which may not align with the goal of …
View article: CoREST in pieces: Dismantling the CoREST complex for cancer therapy and beyond
CoREST in pieces: Dismantling the CoREST complex for cancer therapy and beyond Open
Several landmark studies over the past decade have uncovered a critical role of the CRL3 KBTBD4 ubiquitin ligase complex in regulating stability of corepressor of repressor element 1 silencing transcription factor (CoREST) complex proteins…
View article: KBTBD4-UM171-CoREST1 Axis Prevents Stress-Induced Aging of Human Hematopoietic Stem Cells
KBTBD4-UM171-CoREST1 Axis Prevents Stress-Induced Aging of Human Hematopoietic Stem Cells Open
UM171 is a small molecule degrader of CoREST1 and MYC through KBTBD4 E3 ligase activation. It was initially characterized as a potent hematopoietic stem cell (HSC) self-renewal agonist, with more recent evidence suggesting that it may coun…
View article: Allogeneic Hematopoietic Cell Transplant for B-Cell Lymphomas in the Era of Novel Cellular Therapies: Experience from a Tertiary Canadian Center
Allogeneic Hematopoietic Cell Transplant for B-Cell Lymphomas in the Era of Novel Cellular Therapies: Experience from a Tertiary Canadian Center Open
Background: Allogeneic hematopoietic cell transplant (alloHCT) is a curative option for relapsed/refractory B-cell lymphomas (BCLs), but its role in the evolving field of cellular therapy is increasingly unclear as recent advances in trans…
View article: CD33 Epitope Editing Unlocks UM171-Expanded Cord Blood Grafts for AML Immunotherapy
CD33 Epitope Editing Unlocks UM171-Expanded Cord Blood Grafts for AML Immunotherapy Open
Immunotherapies in acute myeloid leukemia (AML) are limited by shared antigen expression between leukemic and healthy hematopoietic cells, leading to on-target toxicity. Here we developed a clinically scalable strategy to engineer cord blo…
View article: IL1RAP is an immunotherapeutic target for normal karyotype triple-mutated acute myeloid leukemia
IL1RAP is an immunotherapeutic target for normal karyotype triple-mutated acute myeloid leukemia Open
Background Surface antigens of potential clinical significance remain under-characterized in AML. The European Leukemia Network classifies normal karyotype AML (NK-AML) mutated for NPM1 (NPM1c) as a distinct entity associated with favorabl…
View article: DEK::NUP214 acts as an XPO1-dependent transcriptional activator of essential leukemia genes
DEK::NUP214 acts as an XPO1-dependent transcriptional activator of essential leukemia genes Open
The t(6;9)(p22.3;q34.1) translocation/DEK::NUP214 fusion protein defines a distinct subgroup of younger AML patients classified as a separate disease entity by the World Health Organization. DEK is a nuclear factor with multifunctional rol…
View article: Approach to autologous stem cell transplantation in a patient with severe cold agglutinin disease, a case report
Approach to autologous stem cell transplantation in a patient with severe cold agglutinin disease, a case report Open
Background Cold agglutinin disease (CAD) or syndrome (CAS) can be particularly challenging when autologous stem cell transplant (ASCT) is needed. Standard peripheral blood stem cell (PBSC) collection and manipulation involve ex vivo blood …
View article: Deciphering the effect of UM171 on human hematopoietic progenitor cell fate through clonal analysis
Deciphering the effect of UM171 on human hematopoietic progenitor cell fate through clonal analysis Open
Ex vivo expansion of hematopoietic stem cells (HSC) requires the maintenance of a stemness state while cells are proliferating. This can be achieved via exposure to UM171 which leads to the degradation of chromatin modifiers and prevents t…
View article: A Phase II, Open-Label Study of Lenalidomide and Dexamethasone Followed by Donor Lymphocyte Infusions in Relapsed Multiple Myeloma Following Upfront Allogeneic Stem Cell Transplant
A Phase II, Open-Label Study of Lenalidomide and Dexamethasone Followed by Donor Lymphocyte Infusions in Relapsed Multiple Myeloma Following Upfront Allogeneic Stem Cell Transplant Open
Background: To date, the only potential curative treatment for multiple myeloma (MM) remains allogeneic (allo) hematopoietic cell transplant (HCT), although, most patients will eventually relapse. In relapsed patients, donor lymphocyte inf…
View article: UM171 Expands Immature Bone Marrow CD34+ Cells of Patients with Telomere-Biology Disorder
UM171 Expands Immature Bone Marrow CD34+ Cells of Patients with Telomere-Biology Disorder Open
Introduction: Telomere-biology disorders (TBD) are caused by germline defects in genes involved in telomere maintenance, resulting in excessive telomere shortening and limited cell proliferation. Clinically, TBD corresponds to a spectrum o…
View article: Comparative small molecule screening of primary human acute leukemias, engineered human leukemia and leukemia cell lines
Comparative small molecule screening of primary human acute leukemias, engineered human leukemia and leukemia cell lines Open
Targeted therapeutics for high-risk cancers remain an unmet medical need. Here we report the results of a large-scale screen of over 11,000 molecules for their ability to inhibit the survival and growth in vitro of human leukemic cells fro…
View article: A Pilot Study of UM171-Expanded Cord Blood Grafts for Tandem Auto/Allogeneic Hematopoietic Cell Transplant in High and Ultra-High-Risk Myeloma Patients
A Pilot Study of UM171-Expanded Cord Blood Grafts for Tandem Auto/Allogeneic Hematopoietic Cell Transplant in High and Ultra-High-Risk Myeloma Patients Open
Multiple myeloma (MM) remains associated with a poor outcome, particularly in patients with advanced disease and high-risk (HR) cytogenetics. To date, the only curative treatment is allogeneic (allo) hematopoietic cell transplantation (HCT…
View article: Replication of a GWAS signal near<i>HLA-DQA2</i>with acute myeloid leukemia using a disease-only cohort and external population-based controls
Replication of a GWAS signal near<i>HLA-DQA2</i>with acute myeloid leukemia using a disease-only cohort and external population-based controls Open
Acute myeloid leukemia (AML) is the most common type of acute leukemia in adults. Its risk factors include rare and highly penetrant somatic mutations. Genome-wide association studies (GWAS) have also identified four common inherited varia…
View article: UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease
UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease Open
Hematopoietic stem cell (HSC) transplantation with lentiviral vector (LVV)-transduced autologous cells has proven an effective therapeutic strategy for sickle cell disease (SCD). However, ex vivo culture or proliferative stress associated …
View article: Genetic deletion of JAM-C in preleukemic cells rewires leukemic stem cell gene expression program in AML
Genetic deletion of JAM-C in preleukemic cells rewires leukemic stem cell gene expression program in AML Open
The leukemic stem cell (LSC) score LSC-17 based on a stemness–related gene expression signature is an indicator of poor disease outcome in acute myeloid leukemia (AML). However, it is not known whether “niche anchoring” of LSC affects dise…
View article: Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells
Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells Open
Precision gene editing in primary hematopoietic stem and progenitor cells (HSPCs) would facilitate both curative treatments for monogenic disorders as well as disease modelling. Precise efficiencies even with the CRISPR/Cas system, however…
View article: Immunotherapeutic targeting of surfaceome heterogeneity in AML
Immunotherapeutic targeting of surfaceome heterogeneity in AML Open
Immunotherapy remains underexploited in acute myeloid leukemia (AML) compared to other hematological malignancies. Currently, gemtuzumab ozogamicin is the only therapeutic antibody approved for this disease. Here, to identify potential tar…
View article: Reviewer #2 (Public Review): Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells
Reviewer #2 (Public Review): Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells Open
Precision gene editing in primary hematopoietic stem and progenitor cells (HSPCs) would facilitate both curative treatments for monogenic disorders as well as disease modelling. Precise efficiencies even with the CRISPR/Cas system, however…
View article: Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells
Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells Open
Precision gene editing in primary hematopoietic stem and progenitor cells (HSPCs) would facilitate both curative treatments for monogenic disorders as well as disease modelling. Precise efficiencies even with the CRISPR/Cas system, however…
View article: Author response: Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells
Author response: Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells Open
Precision gene editing in primary hematopoietic stem and progenitor cells (HSPCs) would facilitate both curative treatments for monogenic disorders as well as disease modelling. Precise efficiencies even with the CRISPR/Cas system, however…
View article: <i>SF3B1</i> mutations provide genetic vulnerability to copper ionophores in human acute myeloid leukemia
<i>SF3B1</i> mutations provide genetic vulnerability to copper ionophores in human acute myeloid leukemia Open
In a phenotypical screen of 56 acute myeloid leukemia (AML) patient samples and using a library of 10,000 compounds, we identified a hit with increased sensitivity toward SF3B1 -mutated and adverse risk AMLs. Through structure-activity rel…
View article: DELE1 haploinsufficiency causes resistance to mitochondrial stress-induced apoptosis in monosomy 5/del(5q) AML
DELE1 haploinsufficiency causes resistance to mitochondrial stress-induced apoptosis in monosomy 5/del(5q) AML Open
Monosomy 5 and deletions of the chromosome 5q (−5/del(5q)) are recurrent events in de novo adult acute myeloid leukemia (AML), reaching up to 40% of cases in secondary AML. These chromosome anomalies are associated with TP53 mutations and …
View article: The XPO1-FOXC1-HOX Functional Axis Opens New Therapeutic Avenues to Treat DEK-NUP214 AML Patients
The XPO1-FOXC1-HOX Functional Axis Opens New Therapeutic Avenues to Treat DEK-NUP214 AML Patients Open
Introduction: t(6;9)(p23;q34.1)/DEK-NUP214 patients represent a discrete group of younger AML patients recognised as a separate disease entity in the World Health Organization classification of myeloid neoplasms. They typically display a d…