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

PLoS medicine, 2017-09, Vol.14 (9), p.e1002382

2017

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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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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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Gene therapy for sickle cell disease

Hematology, 2023-12, Vol.2023 (1), p.542-547

2023

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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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Treating sickle cell anemia

Science (American Association for the Advancement of Science), 2020-03, Vol.367 (6483), p.1198-1199

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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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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The Lifespan and Turnover of Microglia in the Human Brain

Cell reports (Cambridge), 2017-07, Vol.20 (4), p.779-784

2017

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Outcomes and long-term effects of hematopoietic stem cell transplant in sickle cell disease

Expert review of hematology, 2023-11, Vol.16 (11), p.879-903

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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Gene therapies on the horizon for sickle cell disease: a clinician’s perspective

Expert review of hematology, 2024-09, Vol.17 (9), p.555-566

2024

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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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Hematopoietic Stem Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies

Cell stem cell, 2021-02, Vol.28 (2), p.191-208

2021

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Non‐myeloablative human leukocyte antigen‐matched related donor transplantation in sickle cell disease: outcomes from three independent centres

British journal of haematology, 2021-02, Vol.192 (4), p.761-768

2021

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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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Comparison of CD34+ cells isolated from frozen cord blood and fresh adult peripheral blood of sickle cell disease patients in gene correction of the sickle mutation at late‐stage erythroid differentiation

British journal of haematology, 2021-09, Vol.194 (5), p.e80-e84

2021

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Hematopoietic stem cell mobilization with plerixafor in sickle cell disease

Haematologica (Roma), 2018-05, Vol.103 (5), p.749-750

2018

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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

Volltextzugriff (PDF)

Genetic therapies for the first molecular disease

The Journal of clinical investigation, 2021-04, Vol.131 (8)

2021

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

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

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

Gene therapy for sickle cell disease: An update

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

Genome editing strategies for fetal hemoglobin induction in beta-hemoglobinopathies

Gene therapy for sickle cell disease

Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease

Treating sickle cell anemia

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

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

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

The Lifespan and Turnover of Microglia in the Human Brain

Outcomes and long-term effects of hematopoietic stem cell transplant in sickle cell disease

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

Gene therapies on the horizon for sickle cell disease: a clinician’s perspective

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

Hematopoietic Stem Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies

Non‐myeloablative human leukocyte antigen‐matched related donor transplantation in sickle cell disease: outcomes from three independent centres

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

Comparison of CD34+ cells isolated from frozen cord blood and fresh adult peripheral blood of sickle cell disease patients in gene correction of the sickle mutation at late‐stage erythroid differentiation

Hematopoietic stem cell mobilization with plerixafor in sickle cell disease

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

Genetic therapies for the first molecular disease

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