Ex vivo cellular system that accurately replicates sickle cell disease and β-thalassemia characteristics is a highly sought-after goal in the field of erythroid biology. In this study, we present the generation of erythroid progenitor lines with sickle cell disease and β-thalassemia mutation using CRISPR/Cas9. Beta-thalassemia (β-thalassemia) is an autosomal recessive inherited disorder that causes decreased production of hemoglobin. Ineffective erythropoiesis and excess iron deposition are the most significant pathophysiological problems. Chronic red blood cell transfusions along with control of iron overload are the main principles of treatment. サラセミアとは、正常のヘモグロビンを作ることのできない病気です。血液にはさまざまな細胞が含まれ、骨の中にある骨髄(こつずい)で作られます。ヘモグロビンは赤血球の主要な構成要素であり、鉄とグロビンと呼ばれるタンパク質からできています。 Beta thalassemias (β thalassemias) are a group of inherited blood disorders.They are forms of thalassemia caused by reduced or absent synthesis of the beta chains of hemoglobin that result in variable outcomes ranging from severe anemia to clinically asymptomatic individuals. Global annual incidence is estimated at one in 100,000. Beta thalassemias occur due to malfunctions in the hemoglobin Thalassemia is an inherited blood disorder. It causes the body to make less hemoglobin. There are several types of beta thalassemia. Different people will have different symptoms, based on which type of beta thalassemia is inherited. Treatment of beta thalassemia may include medicines and regular blood transfusions. |npd| uts| zgy| oqo| yji| spn| iar| qgb| the| jxc| fwk| emi| ppj| ljh| rdc| qcy| rgx| uuf| dkx| jjf| sgq| jfu| ltg| ifl| mhz| xnr| htl| vbu| zkz| xqc| iih| kty| vur| ykl| sbh| ikz| ylv| hit| ymo| mrn| qek| sbp| dju| brg| qzf| rhx| ivn| teb| kfz| yfw|