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The New England Journal of Medicine, ISSN 0028-4793, 04/2018, Volume 378, Issue 16, pp. 1479 - 1493
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2017, Volume 25, Issue 5, pp. 1142 - 1154
β-Thalassemia and sickle cell disease (SCD) are the world’s two most widely disseminated hereditary hemoglobinopathies. β-Thalassemia originated in the... 
hematopoietic stem cell | gene therapy | thalassemias | hemoglobinopathies | sickle cell disease | GAMMA-GLOBIN | MEDICINE, RESEARCH & EXPERIMENTAL | ORPHAN NUCLEAR RECEPTORS | HEREDITARY PERSISTENCE | THALASSEMIA MAJOR | CONTROL REGION | GLOBIN GENE | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | FETAL-HEMOGLOBIN | LENTIVIRAL VECTOR | GENETICS & HEREDITY | SICKLE-CELL-DISEASE | HEMATOPOIETIC STEM | Anemia, Sickle Cell - pathology | beta-Thalassemia - pathology | Lentivirus - immunology | Humans | beta-Thalassemia - genetics | Transplantation, Homologous | Lentivirus - genetics | Gammaretrovirus - immunology | Gene Expression | beta-Thalassemia - therapy | Genetic Vectors - chemistry | Genetic Vectors - immunology | Hematopoietic Stem Cell Transplantation | Hematopoietic Stem Cells - metabolism | beta-Globins - genetics | Anemia, Sickle Cell - therapy | Gammaretrovirus - genetics | beta-Globins - metabolism | Anemia, Sickle Cell - metabolism | Hematopoietic Stem Cells - cytology | Gene Editing - methods | HLA Antigens | Mutation | Tissue Donors | beta-Thalassemia - metabolism | Anemia, Sickle Cell - genetics | Genetic Therapy - methods | Transcription factors | Disease | Hematology | Leukocyte migration | Anemia | Thalassemia | Genomes | Transplantation | Gene expression | Patients | Hematopoietic stem cells | Hemopoiesis | Genotype & phenotype | Efficiency | Stem cells | Cell cycle | Hemoglobin | Histocompatibility antigen HLA | Gene therapy | Sickle cell disease | Binding sites | Chronic illnesses | Review
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 03/2017, Volume 376, Issue 9, pp. 848 - 855
Journal Article
Blood, ISSN 0006-4971, 05/2016, Volume 127, Issue 21, pp. 2536 - 2545
Journal Article
Journal Article
Hematology/Oncology Clinics of North America, ISSN 0889-8588, 04/2018, Volume 32, Issue 2, pp. 329 - 342
Journal Article
Molecular Therapy, ISSN 1525-0016, 02/2018, Volume 26, Issue 2, pp. 480 - 495
Although gene transfer to hematopoietic stem cells (HSCs) has shown therapeutic efficacy in recent trials for several individuals with inherited disorders,... 
gene therapy | hematopoietic stem cells | hemoglobin disorders | lentiviral vector | MEDICINE, RESEARCH & EXPERIMENTAL | SUICIDE GENE | HEREDITARY PERSISTENCE | BLOOD PROGENITOR CELLS | TRANSPLANTATION | HIV-1-BASED LENTIVIRAL VECTORS | CYCLOSPORINE-A | VIRAL-DNA | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | FETAL-HEMOGLOBIN | THALASSEMIA | GENETICS & HEREDITY | T-LYMPHOCYTES | Gene Expression | Genes, Transgenic, Suicide | Transduction, Genetic | Humans | Hematopoietic Stem Cell Transplantation | Mice, Transgenic | Hematopoietic Stem Cells - metabolism | beta-Globins - genetics | Genetic Vectors - genetics | Hemoglobinopathies - therapy | Animals | Hemoglobinopathies - genetics | Lentivirus - genetics | ATP Binding Cassette Transporter, Subfamily B - genetics | Mice | Transgenes | Gene Order | Disease Models, Animal | Genetic Therapy - methods | Clinical trials | Herpes simplex | Genomes | Thymidine | Kinases | Suicide | Experiments | Hematopoietic stem cells | Puromycin | MDR1 protein | Hemoglobin | Bone marrow | Acetyltransferase | Sickle cell disease | Thymidine kinase | Cytomegalovirus | Preservation | Therapeutic applications | N-Acetyltransferase | Gene expression | Vectors (Biology) | Contamination | Hereditary diseases | Hemopoiesis | Stem cells | Puromycin N-acetyltransferase | Gene therapy | Life Sciences | Human health and pathology | Biotechnology | Hematology | Original
Journal Article