List of works - Makoto Otsu

A Refined Culture System for Human Induced Pluripotent Stem Cell-Derived Intestinal Epithelial Organoids.

scientific article published in December 2017

A case of C3 deficiency with a novel homozygous two-base deletion in the C3 gene

scientific article published on 01 November 2005

A convenient method for positive selection of retroviral producing cells generating vectors devoid of selectable markers

scientific article

A new red fluorescent protein that allows efficient marking of murine hematopoietic stem cells

scientific article

ACTN1 mutations cause congenital macrothrombocytopenia

scientific article

Adipose Natural Regulatory B Cells Negatively Control Adipose Tissue Inflammation.

scientific article

Characterization of mesenchymal progenitor cells in the crown and root pulp of primary teeth.

scientific article published in January 2015

Comparison of five retrovirus vectors containing the human IL-2 receptor gamma chain gene for their ability to restore T and B lymphocytes in the X-linked severe combined immunodeficiency mouse model.

scientific article published on April 2001

Conditional rod photoreceptor ablation reveals Sall1 as a microglial marker and regulator of microglial morphology in the retina

scientific article published on 26 July 2016

Congenital amegakaryocytic thrombocytopenia iPS cells exhibit defective MPL-mediated signaling

scientific article

Curative haploidentical BMT in a murine model of X-linked chronic granulomatous disease.

scientific article published on 29 April 2015

DNA Methylation Is Involved in the Expression of miR-142-3p in Fibroblasts and Induced Pluripotent Stem Cells.

scientific article published on 2 December 2014

Definitive proof for direct reprogramming of hematopoietic cells to pluripotency.

scientific article

Deregulated intracellular signaling by mutated c-CBL in myeloid neoplasms.

scientific article published on 14 June 2010

Detection of a novel silent deletion, a missense mutation and a nonsense mutation in TCOF1

scientific article published on 01 December 2008

Development of Sendai Virus Vectors and their Potential Applications in Gene Therapy and Regenerative Medicine ⬇️

scientific article published on October 1, 2012

Dissection of Signaling Events Downstream of the c-Mpl Receptor in Murine Hematopoietic Stem Cells Via Motif-Engineered Chimeric Receptors.

scientific article

Effect of donor chimerism to reduce the level of glycosaminoglycans following bone marrow transplantation in a murine model of mucopolysaccharidosis type II.

scientific article published on 13 December 2014

Effects of enzyme replacement therapy on immune function in ADA deficiency patient

scientific article published on 26 June 2015

Flow cytometry analysis of adenosine deaminase (ADA) expression: a simple and reliable tool for the assessment of ADA-deficient patients before and after gene therapy.

scientific article published on February 2002

Frequent pathway mutations of splicing machinery in myelodysplasia

scientific article (publication date: 11 September 2011)

Gain-of-function of mutated C-CBL tumour suppressor in myeloid neoplasms

scientific article published on 20 July 2009

Gene and Cell Therapy for Primary Immunodeficiency Diseases ⬇️

scientific article published on January 1, 2010

Gene therapy: X-SCID transgene leukaemogenicity

scientific article

Generation of Col2a1-EGFP iPS cells for monitoring chondrogenic differentiation

scientific article

Generation of engraftable hematopoietic stem cells from induced pluripotent stem cells by way of teratoma formation.

scientific article

Generation of induced pluripotent stem cells derived from primary and secondary myelofibrosis patient samples

scientific article published on 20 May 2014

Generation of induced pluripotent stem cells from primary chronic myelogenous leukemia patient samples

scientific article published on 16 May 2012

Generation of three induced pluripotent stem cell lines from postmortem tissue derived following sudden death of a young patient with STXBP1 mutation

scientific article published on 18 June 2019

Generation of transgenic mouse line expressing Kusabira Orange throughout body, including erythrocytes, by random segregation of provirus method

scientific article

Growth promotion of genetically modified hematopoietic progenitors using an antibody/c-Mpl chimera.

scientific article published on 22 June 2011

Haploinsufficiency of Sf3b1 leads to compromised stem cell function but not to myelodysplasia

scientific article published on 18 February 2014

Hes1 promotes blast crisis in chronic myelogenous leukemia through MMP-9 upregulation in leukemic cells.

scientific article

Heterozygous ITGA2B R995W mutation inducing constitutive activation of the αIIbβ3 receptor affects proplatelet formation and causes congenital macrothrombocytopenia

scientific article

Immortalization of erythroblasts by c-MYC and BCL-XL enables large-scale erythrocyte production from human pluripotent stem cells

scientific article

In vivo competitive studies between normal and common gamma chain-defective bone marrow cells: implications for gene therapy

scientific article published in September 2000

In vivo imaging visualizes discoid platelet aggregations without endothelium disruption and implicates contribution of inflammatory cytokine and integrin signaling

scientific article

Intracellular estrogen receptor-binding fragment-associated antigen 9 exerts in vivo tumor-promoting effects via its coiled-coil region.

scientific article published on 3 May 2011

In vitro cell subtype-specific transduction of adeno-associated virus in mouse and marmoset retinal explant culture

scientific article published on 21 August 2012

Kinetics and effect of integrin expression on human CD34(+) cells during murine leukemia virus-derived retroviral transduction with recombinant fibronectin for stem cell gene therapy

scientific article published in July 2009

Lack of dominant-negative effects of a truncated gamma(c) on retroviral-mediated gene correction of immunodeficient mice.

scientific article

Minimum requirement of donor cells to reduce the glycolipid storage following bone marrow transplantation in a murine model of Fabry disease

scientific article published in May 2011

Multiple allogeneic progenitors in combination function as a unit to support early transient hematopoiesis in transplantation

scientific article published on 08 August 2016

Non-myeloablative preconditioning with ACK2 (anti-c-kit antibody) is efficient in bone marrow transplantation for murine models of mucopolysaccharidosis type II.

scientific article published on 21 August 2016

Nuclear receptor gene alteration in human induced pluripotent stem cells with hepatic differentiation propensity.

scientific article published on 6 May 2014

Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning

scientific article published on 15 April 2015

Potent vaccine therapy with dendritic cells genetically modified by the gene-silencing-resistant retroviral vector GCDNsap

scientific article

Profiling of microRNA in human and mouse ES and iPS cells reveals overlapping but distinct microRNA expression patterns

scientific article

Reciprocal Inflammatory Signaling Between Intestinal Epithelial Cells and Adipocytes in the Absence of Immune Cells.

scientific article published on 2 August 2017

Recurrent mutations in multiple components of the cohesin complex in myeloid neoplasms

scientific article

Reduction of N-glycolylneuraminic acid in human induced pluripotent stem cells generated or cultured under feeder- and serum-free defined conditions

scientific article

Robust and highly efficient hiPSC generation from patient non-mobilized peripheral blood-derived CD34+ cells using the auto-erasable Sendai virus vector

scientific article published on 24 June 2019

Screening of drugs to treat 8p11 myeloproliferative syndrome using patient-derived induced pluripotent stem cells with fusion gene CEP110-FGFR1

scientific article published on 24 March 2015

Somatic mosaicism in Wiskott--Aldrich syndrome suggests in vivo reversion by a DNA slippage mechanism.

scientific article published on 10 July 2001

Stable transgene expression in mice generated from retrovirally transduced embryonic stem cells

scientific article

Stage-specific roles for CXCR4 signaling in murine hematopoietic stem/progenitor cells in the process of bone marrow repopulation

scientific article published in July 2014

Stem cell therapy: an exercise in patience and prudence ⬇️

scientific article published on January 5, 2013

Successful intravenous immunoglobulin therapy for recurrent pneumococcal otitis media in young children

scientific article published on 01 March 1994

T cell growth control using hapten-specific antibody/interleukin-2 receptor chimera.

scientific article

The actin polymerization regulator WAVE2 is required for early bone marrow repopulation by hematopoietic stem cells.

scientific article published on May 2009

The generation of induced pluripotent stem cells (iPSCs) from patients with infantile and late-onset types of Pompe disease and the effects of treatment with acid-α-glucosidase in Pompe's iPSCs

scientific article published on 4 March 2014

Top-down motif engineering of a cytokine receptor for directing ex vivo expansion of hematopoietic stem cells

scientific article published on 23 September 2013

Transient activation of c-MYC expression is critical for efficient platelet generation from human induced pluripotent stem cells

scientific article

Using patient-derived iPSCs to develop humanized mouse models for chronic myelomonocytic leukemia and therapeutic drug identification, including liposomal clodronate

scientific article published in Scientific Reports

[Patient-derived iPS cells as a tool for gene therapy research]

scientific article published on 01 August 2015

List of works by Makoto Otsu

A Refined Culture System for Human Induced Pluripotent Stem Cell-Derived Intestinal Epithelial Organoids.

A case of C3 deficiency with a novel homozygous two-base deletion in the C3 gene

A convenient method for positive selection of retroviral producing cells generating vectors devoid of selectable markers

A new red fluorescent protein that allows efficient marking of murine hematopoietic stem cells

ACTN1 mutations cause congenital macrothrombocytopenia

Adipose Natural Regulatory B Cells Negatively Control Adipose Tissue Inflammation.

Characterization of mesenchymal progenitor cells in the crown and root pulp of primary teeth.

Comparison of five retrovirus vectors containing the human IL-2 receptor gamma chain gene for their ability to restore T and B lymphocytes in the X-linked severe combined immunodeficiency mouse model.

Conditional rod photoreceptor ablation reveals Sall1 as a microglial marker and regulator of microglial morphology in the retina

Congenital amegakaryocytic thrombocytopenia iPS cells exhibit defective MPL-mediated signaling

Curative haploidentical BMT in a murine model of X-linked chronic granulomatous disease.

DNA Methylation Is Involved in the Expression of miR-142-3p in Fibroblasts and Induced Pluripotent Stem Cells.

Definitive proof for direct reprogramming of hematopoietic cells to pluripotency.

Deregulated intracellular signaling by mutated c-CBL in myeloid neoplasms.

Detection of a novel silent deletion, a missense mutation and a nonsense mutation in TCOF1

Development of Sendai Virus Vectors and their Potential Applications in Gene Therapy and Regenerative Medicine ⬇️

Dissection of Signaling Events Downstream of the c-Mpl Receptor in Murine Hematopoietic Stem Cells Via Motif-Engineered Chimeric Receptors.

Effect of donor chimerism to reduce the level of glycosaminoglycans following bone marrow transplantation in a murine model of mucopolysaccharidosis type II.

Effects of enzyme replacement therapy on immune function in ADA deficiency patient

Flow cytometry analysis of adenosine deaminase (ADA) expression: a simple and reliable tool for the assessment of ADA-deficient patients before and after gene therapy.

Frequent pathway mutations of splicing machinery in myelodysplasia

Gain-of-function of mutated C-CBL tumour suppressor in myeloid neoplasms

Gene and Cell Therapy for Primary Immunodeficiency Diseases ⬇️

Gene therapy: X-SCID transgene leukaemogenicity

Generation of Col2a1-EGFP iPS cells for monitoring chondrogenic differentiation

Generation of engraftable hematopoietic stem cells from induced pluripotent stem cells by way of teratoma formation.

Generation of induced pluripotent stem cells derived from primary and secondary myelofibrosis patient samples

Generation of induced pluripotent stem cells from primary chronic myelogenous leukemia patient samples

Generation of three induced pluripotent stem cell lines from postmortem tissue derived following sudden death of a young patient with STXBP1 mutation

Generation of transgenic mouse line expressing Kusabira Orange throughout body, including erythrocytes, by random segregation of provirus method

Growth promotion of genetically modified hematopoietic progenitors using an antibody/c-Mpl chimera.

Haploinsufficiency of Sf3b1 leads to compromised stem cell function but not to myelodysplasia

Hes1 promotes blast crisis in chronic myelogenous leukemia through MMP-9 upregulation in leukemic cells.

Heterozygous ITGA2B R995W mutation inducing constitutive activation of the αIIbβ3 receptor affects proplatelet formation and causes congenital macrothrombocytopenia

Immortalization of erythroblasts by c-MYC and BCL-XL enables large-scale erythrocyte production from human pluripotent stem cells

In vivo competitive studies between normal and common gamma chain-defective bone marrow cells: implications for gene therapy

In vivo imaging visualizes discoid platelet aggregations without endothelium disruption and implicates contribution of inflammatory cytokine and integrin signaling

Intracellular estrogen receptor-binding fragment-associated antigen 9 exerts in vivo tumor-promoting effects via its coiled-coil region.

In vitro cell subtype-specific transduction of adeno-associated virus in mouse and marmoset retinal explant culture

Kinetics and effect of integrin expression on human CD34(+) cells during murine leukemia virus-derived retroviral transduction with recombinant fibronectin for stem cell gene therapy

Lack of dominant-negative effects of a truncated gamma(c) on retroviral-mediated gene correction of immunodeficient mice.

Minimum requirement of donor cells to reduce the glycolipid storage following bone marrow transplantation in a murine model of Fabry disease

Multiple allogeneic progenitors in combination function as a unit to support early transient hematopoiesis in transplantation

Non-myeloablative preconditioning with ACK2 (anti-c-kit antibody) is efficient in bone marrow transplantation for murine models of mucopolysaccharidosis type II.

Nuclear receptor gene alteration in human induced pluripotent stem cells with hepatic differentiation propensity.

Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning

Potent vaccine therapy with dendritic cells genetically modified by the gene-silencing-resistant retroviral vector GCDNsap

Profiling of microRNA in human and mouse ES and iPS cells reveals overlapping but distinct microRNA expression patterns

Reciprocal Inflammatory Signaling Between Intestinal Epithelial Cells and Adipocytes in the Absence of Immune Cells.

Recurrent mutations in multiple components of the cohesin complex in myeloid neoplasms

Reduction of N-glycolylneuraminic acid in human induced pluripotent stem cells generated or cultured under feeder- and serum-free defined conditions

Robust and highly efficient hiPSC generation from patient non-mobilized peripheral blood-derived CD34+ cells using the auto-erasable Sendai virus vector

Screening of drugs to treat 8p11 myeloproliferative syndrome using patient-derived induced pluripotent stem cells with fusion gene CEP110-FGFR1

Somatic mosaicism in Wiskott--Aldrich syndrome suggests in vivo reversion by a DNA slippage mechanism.

Stable transgene expression in mice generated from retrovirally transduced embryonic stem cells

Stage-specific roles for CXCR4 signaling in murine hematopoietic stem/progenitor cells in the process of bone marrow repopulation

Stem cell therapy: an exercise in patience and prudence ⬇️

Successful intravenous immunoglobulin therapy for recurrent pneumococcal otitis media in young children

T cell growth control using hapten-specific antibody/interleukin-2 receptor chimera.

The actin polymerization regulator WAVE2 is required for early bone marrow repopulation by hematopoietic stem cells.

The generation of induced pluripotent stem cells (iPSCs) from patients with infantile and late-onset types of Pompe disease and the effects of treatment with acid-α-glucosidase in Pompe's iPSCs

Top-down motif engineering of a cytokine receptor for directing ex vivo expansion of hematopoietic stem cells

Transient activation of c-MYC expression is critical for efficient platelet generation from human induced pluripotent stem cells

Using patient-derived iPSCs to develop humanized mouse models for chronic myelomonocytic leukemia and therapeutic drug identification, including liposomal clodronate

[Patient-derived iPS cells as a tool for gene therapy research]