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Objective(s):β- thalassemia is one of the most common genetic disorders in the world. As one of the promising treatment strategies, fetal hemoglobin (Hb F) can be induced. The present study ...
Reactivation of γ-globin expression is a promising therapeutic approach for β-hemoglobinopathies. Here, we propose a novel Cas9/AAV6-mediated genome editing strategy for the treatment of β ...
Abstract Sickle cell anemia and thalassemia constitute the most common genetic diseases in the world. Affected patients carry a heavy burden of morbidity and early mortality. With improved ...
for suppressing BCL11A gene expression are promising treatment options for hemoglobinopathies (49). In addition, editing approaches could be theoretically safer compared to LVs integrating semi-randomly ...
Abstract Beta- thalassemia is one of the most common monogenic disorders. Standard treatment of the most severe forms, i.e., transfusion-dependent thalassemia (TDT) with long-term transfusion and iron ...
and delivery of novel gene therapies for genetic disease. Compared with earlier technologies, the system offers greater precision and ease of use and offers promise for both ex vivo manipulation of somatic cells ...
Abstract The therapeutic use of patient-specific induced pluripotent stem cells (iPSCs) is emerging as a potential treatment of β- thalassemia . Ideally, patient-specific iPSCs would be genetically ...
RN , Lee CM, Bao G. Treating hemoglobinopathies using gene-correction approaches : Promises and challenges . Hum Genet 2016 ; 135 : 993 – 1010 . Google Scholar Crossref Search ADS PubMed WorldCat 7 Yu ...
the effect of this drug. Currently, different chelation-therapy regimens yield relatively similar results and are still considered the most promising treatment for iron overload in patients with TM (Table 3 ...
that promises more targeted and tailored therapeutic solutions. Among the advances are new therapeutic targets to raise production of fetal hemoglobin, which eases the symptoms of the disease; new approaches ...
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