Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
Copyright policy )Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
Preclinical efficacy and safety data in mice provide support for ex vivo β-globin gene correction to treat patients with sickle cell disease.
- Rice University United States
- National Institute of Health Pakistan
- Integrated DNA Technologies (United States) United States
- APC Microbiome Institute Ireland
- Stanford University School of Medicine United States
Medical Biotechnology, EFFICIENT, beta-Globins, Regenerative Medicine, THERAPY, Medical and Health Sciences, Mice, DOUBLE-STRANDED BREAKS, Stem Cell Research - Nonembryonic - Human, Heterocyclic Compounds, Cardiovascular Medicine and Haematology, HEMOGLOBIN, CURE, Gene Editing, CRISPR-Cas Systems/genetics, Anemia, Gene Therapy, Hematology, Biological Sciences, Hematopoietic Stem Cell Mobilization, Sickle Cell, Blood, Biomedical engineering, Biotechnology, EXPRESSION, Medical biotechnology, Anemia, Sickle Cell, Rare Diseases, Clinical Research, Genetics, Animals, Humans, Sickle Cell Disease, Transplantation, Biomedical and Clinical Sciences, 5.2 Cellular and gene therapies, Reproducibility of Results, Stem Cell Research, Hematopoietic Stem Cells, BONE-MARROW-TRANSPLANTATION, Anemia, Sickle Cell/genetics, beta-Globins/genetics, Orphan Drug, Good Health and Well Being, PROGENITOR CELLS, CRISPR-Cas Systems, MIXED CHIMERISM
Medical Biotechnology, EFFICIENT, beta-Globins, Regenerative Medicine, THERAPY, Medical and Health Sciences, Mice, DOUBLE-STRANDED BREAKS, Stem Cell Research - Nonembryonic - Human, Heterocyclic Compounds, Cardiovascular Medicine and Haematology, HEMOGLOBIN, CURE, Gene Editing, CRISPR-Cas Systems/genetics, Anemia, Gene Therapy, Hematology, Biological Sciences, Hematopoietic Stem Cell Mobilization, Sickle Cell, Blood, Biomedical engineering, Biotechnology, EXPRESSION, Medical biotechnology, Anemia, Sickle Cell, Rare Diseases, Clinical Research, Genetics, Animals, Humans, Sickle Cell Disease, Transplantation, Biomedical and Clinical Sciences, 5.2 Cellular and gene therapies, Reproducibility of Results, Stem Cell Research, Hematopoietic Stem Cells, BONE-MARROW-TRANSPLANTATION, Anemia, Sickle Cell/genetics, beta-Globins/genetics, Orphan Drug, Good Health and Well Being, PROGENITOR CELLS, CRISPR-Cas Systems, MIXED CHIMERISM
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