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Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

Nature medicine, 2021-04, Vol.27 (4), p.677-687

2021

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Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB‐206 study

American journal of hematology, 2023-01, Vol.98 (1), p.11-22

2023

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Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixafor

Haematologica (Roma), 2020-10, Vol.105 (10), p.e497

2020

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Evaluation of genetic diversity using 31 microsatellites in Miyako horses

Journal of Equine Science, 2023, Vol.34(1), pp.1-6

2023

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Prostaglandin E2 Enhances Human Cord Blood Stem Cell Xenotransplants and Shows Long-Term Safety in Preclinical Nonhuman Primate Transplant Models

Cell stem cell, 2011-04, Vol.8 (4), p.445-458

2011

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Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders

Nature communications, 2019-10, Vol.10 (1), p.4479-14, Article 4479

2019

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Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model

Nature communications, 2023-10, Vol.14 (1), p.6291-6291, Article 6291

2023

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Genome editing of HBG1 and HBG2 to induce fetal hemoglobin

Blood advances, 2019-11, Vol.3 (21), p.3379-3392

2019

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Biallelic correction of sickle cell disease‐derived induced pluripotent stem cells (iPSCs) confirmed at the protein level through serum‐free iPS‐sac/erythroid differentiation

Stem cells translational medicine, 2020-05, Vol.9 (5), p.590-602

2020

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Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease

Molecular therapy. Methods & clinical development, 2021-06, Vol.21, p.121-132

2021

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Immunoresponse to Gene-Modified Hematopoietic Stem Cells

Molecular therapy. Methods & clinical development, 2020-03, Vol.16, p.42-49

2020

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Path to the Clinic: Assessment of iPSC-Based Cell Therapies In Vivo in a Nonhuman Primate Model

Cell reports (Cambridge), 2014-05, Vol.7 (4), p.1298-1309

2014

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Hematopoietic Stem Cell-Targeted Gene Therapy Using Lentiviral Vectors

Nihon Ika Daigaku Igakkai Zasshi, 2023/08/31, Vol.19(3), pp.205-210

Uchida, Naoya

2023

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Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease

Cells (Basel, Switzerland), 2022-06, Vol.11 (11), p.1843

2022

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High-Efficiency Lentiviral Transduction of Human CD34+ Cells in High-Density Culture with Poloxamer and Prostaglandin E2

Molecular therapy. Methods & clinical development, 2019-06, Vol.13, p.187-196

2019

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Vasopressin stimulates the proliferation and differentiation of red blood cell precursors and improves recovery from anemia

Science translational medicine, 2017-11, Vol.9 (418)

2017

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Low-Dose Busulfan Reduces Human CD34+ Cell Doses Required for Engraftment in c-kit Mutant Immunodeficient Mice

Molecular therapy. Methods & clinical development, 2019-12, Vol.15, p.430-437

2019

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MASL1 induces erythroid differentiation in human erythropoietin-dependent CD34+ cells through the Raf/MEK/ERK pathway

Blood, 2013-04, Vol.121 (16), p.3216-3227

2013

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Forced enhancer-promoter rewiring to alter gene expression in animal models

Molecular therapy. Nucleic acids, 2023-03, Vol.31, p.452-465

2023

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βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells

Molecular therapy. Methods & clinical development, 2020-06, Vol.17, p.912-921

2020

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Correction to: Definitive hematopoietic stem/progenitor cells from human embryonic stem cells through serum/feeder-free organoid-induced differentiation

Stem cell research & therapy, 2020-12, Vol.11 (1), p.544-544, Article 544

2020

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Pre-existing immunity does not impair the engraftment of CRISPR-Cas9-edited cells in rhesus macaques conditioned with busulfan or radiation

Molecular therapy. Methods & clinical development, 2023-06, Vol.29, p.483-493

2023

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Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models

Cell reports. Medicine, 2021-04, Vol.2 (4), p.100247, Article 100247

2021

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Decitabine suspends human CD34+ cell differentiation and proliferation during lentiviral transduction

PloS one, 2014-08, Vol.9 (8), p.e104022

2014

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Development of a Human Immunodeficiency Virus Type 1-Based Lentiviral Vector That Allows Efficient Transduction of both Human and Rhesus Blood Cells

Journal of Virology, 2009-10, Vol.83 (19), p.9854-9862

2009

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Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB‐206 study

Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixafor

Evaluation of genetic diversity using 31 microsatellites in Miyako horses

Prostaglandin E2 Enhances Human Cord Blood Stem Cell Xenotransplants and Shows Long-Term Safety in Preclinical Nonhuman Primate Transplant Models

Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders

Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model

Genome editing of HBG1 and HBG2 to induce fetal hemoglobin

Biallelic correction of sickle cell disease‐derived induced pluripotent stem cells (iPSCs) confirmed at the protein level through serum‐free iPS‐sac/erythroid differentiation

Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease

Immunoresponse to Gene-Modified Hematopoietic Stem Cells

Path to the Clinic: Assessment of iPSC-Based Cell Therapies In Vivo in a Nonhuman Primate Model

Hematopoietic Stem Cell-Targeted Gene Therapy Using Lentiviral Vectors

Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease

High-Efficiency Lentiviral Transduction of Human CD34+ Cells in High-Density Culture with Poloxamer and Prostaglandin E2

Vasopressin stimulates the proliferation and differentiation of red blood cell precursors and improves recovery from anemia

Low-Dose Busulfan Reduces Human CD34+ Cell Doses Required for Engraftment in c-kit Mutant Immunodeficient Mice

MASL1 induces erythroid differentiation in human erythropoietin-dependent CD34+ cells through the Raf/MEK/ERK pathway

Forced enhancer-promoter rewiring to alter gene expression in animal models

βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells

Correction to: Definitive hematopoietic stem/progenitor cells from human embryonic stem cells through serum/feeder-free organoid-induced differentiation

Pre-existing immunity does not impair the engraftment of CRISPR-Cas9-edited cells in rhesus macaques conditioned with busulfan or radiation

Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models

Decitabine suspends human CD34+ cell differentiation and proliferation during lentiviral transduction

Development of a Human Immunodeficiency Virus Type 1-Based Lentiviral Vector That Allows Efficient Transduction of both Human and Rhesus Blood Cells

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