Modeling Transient Abnormal Myelopoiesis Using Induced Pluripotent Stem Cells and CRISPR/Cas9 Technology

Mechanisms of Progression of Myeloid Preleukemia to Transformed Myeloid Leukemia in Children with Down Syndrome

(2019) Maurice Labuhn et al.   CANCER CELL

Chromatin occupancy and epigenetic analysis reveal new insights into the function of GATA1 N-terminus in erythropoiesis

(2019) Te Ling et al.   BLOOD

CRISPOR: intuitive guide selection for CRISPR/Cas9 genome editing experiments and screens

(2018) Jean-Paul Concordet et al.   NUCLEIC ACIDS RESEARCH

CRISPR/Cas9-Directed Reassignment of the GATA1 Initiation Codon in K562 Cells to Recapitulate AML in Down Syndrome

(2017) Kevin M. Bloh et al.   Molecular Therapy-Nucleic Acids

Evolution of myeloid leukemia in children with Down syndrome

(2016) Satoshi Saida   INTERNATIONAL JOURNAL OF HEMATOLOGY

Systematic Cellular Disease Models Reveal Synergistic Interaction of Trisomy 21 and GATA1 Mutations in Hematopoietic Abnormalities

(2016) Kimihiko Banno et al.   Cell Reports

The biology of pediatric acute megakaryoblastic leukemia

(2015) T. A. Gruber et al.   BLOOD

Pluripotent stem cells reveal erythroid-specific activities of the GATA1 N-terminus

(2015) Marta Byrska-Bishop et al.   JOURNAL OF CLINICAL INVESTIGATION

Acute myeloid leukemia in children and adolescents: identification of new molecular targets brings promise of new therapies

(2015) E. A. Kolb et al.   Hematology-American Society of Hematology Education Program

A mutation in the Kozak sequence ofGATA4hampers translation in a family with atrial septal defects

(2014) Rajiv A. Mohan et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Easy quantitative assessment of genome editing by sequence trace decomposition

(2014) Eva K. Brinkman et al.   NUCLEIC ACIDS RESEARCH

Role of astroglia in Down’s syndrome revealed by patient-derived human-induced pluripotent stem cells

(2014) Chen Chen et al.   Nature Communications

Down syndrome-associated haematopoiesis abnormalities created by chromosome transfer and genome editing technologies

(2014) Yasuhiro Kazuki et al.   Scientific Reports

Exome sequencing identifies putative drivers of progression of transient myeloproliferative disorder to AMKL in infants with Down syndrome

(2013) S. I. Nikolaev et al.   BLOOD

GATA1s induces hyperproliferation of eosinophil precursors in Down syndrome transient leukemia

(2013) A Maroz et al.   LEUKEMIA

The landscape of somatic mutations in Down syndrome–related myeloid disorders

(2013) Kenichi Yoshida et al.   NATURE GENETICS

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Generation of Megakaryocytic Progenitors from Human Embryonic Stem Cells in a Feeder- and Serum-Free Medium

(2013) Marjorie Pick et al.   PLoS One

Promise and challenges of human iPSC-based hematologic disease modeling and treatment

(2012) Zhaohui Ye et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY

Trisomy 21-associated defects in human primitive hematopoiesis revealed through induced pluripotent stem cells

(2012) S. T. Chou et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Altered hematopoiesis in trisomy 21 as revealed through in vitro differentiation of isogenic human pluripotent cells

(2012) G. A. MacLean et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

iPSC Disease Modeling

(2012) F. Soldner et al.   SCIENCE

Down syndrome and GATA1 mutations in transient abnormal myeloproliferative disorder: mutation classes correlate with progression to myeloid leukemia

(2010) R. Kanezaki et al.   BLOOD

Developmental stage-specific interplay of GATA1 and IGF signaling in fetal megakaryopoiesis and leukemogenesis

(2010) J.-H. Klusmann et al.   GENES & DEVELOPMENT

Acute leukemia in children with Down syndrome

(2010) A. C. Xavier et al.   HAEMATOLOGICA

Trisomic dose of several chromosome 21 genes perturbs haematopoietic stem and progenitor cell differentiation in Down's syndrome

(2010) S De Vita et al.   ONCOGENE

Insights into the manifestations, outcomes, and mechanisms of leukemogenesis in Down syndrome

(2009) S. Malinge et al.   BLOOD

Induction of hyperproliferative fetal megakaryopoiesis by an N-terminally truncated GATA1 mutant

(2009) Ritsuko Shimizu et al.   GENES TO CELLS

Direct Binding of pRb/E2F-2 to GATA-1 Regulates Maturation and Terminal Cell Division during Erythropoiesis

(2009) Zahra Kadri et al.   PLOS BIOLOGY

Abnormalities in the myeloid progenitor compartment in Down syndrome fetal liver precede acquisition of GATA1 mutations

(2008) O. Tunstall-Pedoe et al.   BLOOD

Trisomy 21 enhances human fetal erythro-megakaryocytic development

(2008) S. T. Chou et al.   BLOOD

Disease-Specific Induced Pluripotent Stem Cells

(2008) In-Hyun Park et al.   CELL