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Sickle cell disease (SCD) is one of the most common life-threatening monogenic diseases affecting millions of people worldwide. Allogenic hematopietic stem cell transplantation is the only known cure for the disease with high success rates, but the limited availability of matched sibling donors and the high risk of transplantation-related side effects force the scientific community to envision additional therapies. Ex vivo gene therapy through globin gene addition has been investigated extensively and is currently being tested in clinical trials that have begun reporting encouraging data. Recent improvements in our understanding of the molecular pathways controlling mammalian erythropoiesis and globin switching offer new and exciting therapeutic options. Rapid and substantial advances in genome engineering tools, particularly CRISPR/Cas9, have raised the possibility of genetic correction in induced pluripotent stem cells as well as patient-derived hematopoietic stem and progenitor cells. However, these techniques are still in their infancy, and safety/efficacy issues remain that must be addressed before translating these promising techniques into clinical practice. 相似文献
494.
《Molecular cell》2023,83(10):1640-1658.e9
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495.
Jolanta Rzymowska 《Biological trace element research》1996,53(1-3):235-239
In this study, magnesium and iron concentrations were measured in lymphocytes from patients with acute lymphoblastic leukemia
(ALL), acute myeloid leukemia (AML), and hemolytic anemia (HA) before and after chemotherapy treatment. The results were compared
with those of control subjects. Magnesium concentrations were significantly lower in the patient groups, compared with control
values. However, no significant differences, except in the HA group, were found among magnesium concentrations of the patient
groups them-selves. Iron level values were at physiological range in all groups. Similarly, no statistically significant differences
were found between lymphocyte magnesium concentrations before and after chemotherapy treatment in the patient groups. Fe+3 values were higher in the ALL and HA groups with respect to the group before chemotherapy. 相似文献