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Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease

The New England journal of medicine, 2022-02, Vol.386 (7), p.617-628

2022

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At least 20% donor myeloid chimerism is necessary to reverse the sickle phenotype after allogeneic HSCT

Blood, 2017-10, Vol.130 (17), p.1946-1948

2017

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Hematopoietic stem cells from pluripotent stem cells: Clinical potential, challenges, and future perspectives

Stem cells translational medicine, 2020-12, Vol.9 (12), p.1549-1557

2020

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Stem cell transplantation in sickle cell disease: therapeutic potential and challenges faced

Expert review of hematology, 2018-07, Vol.11 (7), p.547-565

2018

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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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Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now

Blood, 2011-08, Vol.118 (5), p.1197-1207

2011

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Toward effective hematopoietic stem cell gene therapies: Optimized conditioning regimen and stem cell source in harmony

Molecular therapy. Methods & clinical development, 2023-09, Vol.30, p.411-412

2023

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Hematopoietic stem cell mobilization with plerixafor in sickle cell disease

Haematologica (Roma), 2018-05, Vol.103 (5), p.749

2018

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Definitive Erythropoiesis from Pluripotent Stem Cells: Recent Advances and Perspectives

Cell Biology and Translational Medicine, Volume 3, 2018-01, Vol.1107, p.1-13

2018

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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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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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A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease

Molecular therapy, 2021-04, Vol.29 (4), p.1355-1356

2021

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Prevalence of neutropenia in the U.S. population: age, sex, smoking status, and ethnic differences

Annals of internal medicine, 2007-04, Vol.146 (7), p.486

2007

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CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease

Molecular therapy. Methods & clinical development, 2021-12, Vol.23, p.276-285

2021

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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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Hydroxyurea-Increased Fetal Hemoglobin Is Associated with Less Organ Damage and Longer Survival in Adults with Sickle Cell Anemia

PloS one, 2015-11, Vol.10 (11), p.e0141706

2015

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Gene therapy for sickle cell disease: An update

Cytotherapy (Oxford, England), 2018-07, Vol.20 (7), p.899-910

2018

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A new frontier: FDA approvals for gene therapy in sickle cell disease

Molecular therapy, 2024-02, Vol.32 (2), p.264-267

2024

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Wild-type macrophages reverse disease in heme oxygenase 1-deficient mice

Blood, 2014-08, Vol.124 (9), p.1522-1530

2014

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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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Gene therapy for sickle cell disease: moving from the bench to the bedside

Blood, 2021-09, Vol.138 (11), p.932-941

2021

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Genome editing strategies for fetal hemoglobin induction in beta-hemoglobinopathies

Human molecular genetics, 2020-09, Vol.29 (R1), p.R100-R106

2020

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Robust generation of erythroid and multilineage hematopoietic progenitors from human iPSCs using a scalable monolayer culture system

Stem cell research, 2019-12, Vol.41, p.101600-101600, Article 101600

2019

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Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

Oral tetrahydrouridine and decitabine for non-cytotoxic epigenetic gene regulation in sickle cell disease: A randomized phase 1 study

At least 20% donor myeloid chimerism is necessary to reverse the sickle phenotype after allogeneic HSCT

Hematopoietic stem cells from pluripotent stem cells: Clinical potential, challenges, and future perspectives

Stem cell transplantation in sickle cell disease: therapeutic potential and challenges faced

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

Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now

Toward effective hematopoietic stem cell gene therapies: Optimized conditioning regimen and stem cell source in harmony

Hematopoietic stem cell mobilization with plerixafor in sickle cell disease

Definitive Erythropoiesis from Pluripotent Stem Cells: Recent Advances and Perspectives

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

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

A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease

Prevalence of neutropenia in the U.S. population: age, sex, smoking status, and ethnic differences

CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease

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

Hydroxyurea-Increased Fetal Hemoglobin Is Associated with Less Organ Damage and Longer Survival in Adults with Sickle Cell Anemia

Gene therapy for sickle cell disease: An update

A new frontier: FDA approvals for gene therapy in sickle cell disease

Wild-type macrophages reverse disease in heme oxygenase 1-deficient mice

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

Gene therapy for sickle cell disease: moving from the bench to the bedside

Genome editing strategies for fetal hemoglobin induction in beta-hemoglobinopathies

Robust generation of erythroid and multilineage hematopoietic progenitors from human iPSCs using a scalable monolayer culture system

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