2022
DOI: 10.1080/14728222.2022.2066519
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Targeting fetal hemoglobin expression to treat β hemoglobinopathies

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Cited by 10 publications
(9 citation statements)
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“…Beta Thalassemia more than 200 mutations in the beta globin gene have been identified as producing beta thalassemia worldwide. beta thalassemia is caused by mutation on chromosome 11, Unlike the deletion that create most of the alpha thalassemia syndromes that affect all aspect of beta globin formation : stability of the beta globin production, transcription and translation 25 .…”
Section: Discussionmentioning
confidence: 99%
“…Beta Thalassemia more than 200 mutations in the beta globin gene have been identified as producing beta thalassemia worldwide. beta thalassemia is caused by mutation on chromosome 11, Unlike the deletion that create most of the alpha thalassemia syndromes that affect all aspect of beta globin formation : stability of the beta globin production, transcription and translation 25 .…”
Section: Discussionmentioning
confidence: 99%
“…Laboratory measures including total hemoglobin and Hb electrophoresis, assessing the number of genetically targeted cells, markers of ineffective erythropoiesis, and assessments of iron overload ( including improvement in ineffective erythropoiesis. 64,65 Measurements of HbF concentration and F-cell expression are thus important endpoints for establishing the effectiveness of gene editing therapies 50 (Table 1).…”
Section: Laboratory and Radiologic Endpointsmentioning
confidence: 99%
“…Moreover, β-thalassemic infants show symptoms after attenuating HbF production. Recently, researchers have verified the effect of high HbF synthesis on the improvement of clinical symptoms of β-thalassemic patients [ 37 , 38 , 39 ]. All together, these observations lead to further investigation of HbF inducers with emphasize on the role of naturally higher levels of HbF in β-thalassemia patients [ 40 , 41 ].…”
Section: Hbf or Gamma Globin Induction Using Gene Editing Toolsmentioning
confidence: 99%