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Sickle Cell Disease: Historical Overview and Current Therapies
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DOI：10.1002/pd.70195.png)
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Abstract
En 中文
Sickle cell disease (SCD) affects millions worldwide, yet the limited treatment options currently available do not always adequately control the disease and carry significant side effects. At present, the only curative treatment is hematopoietic stem cell (HSC) transplantation, a procedure that carries considerable challenges and numerous risks to the patient, due in large part to the toxic conditioning regimens needed to obtain sufficient donor HSC engraftment. Thus, there is an urgent unmet medical need for novel SCD therapies that promise a cure for this devastating disease following a single treatment. Furthermore, while existing treatments often only commence in late childhood or adolescence, the clinical manifestations of SCD start during early infancy, placing the child at risk of permanent organ damage and potentially life-threatening complications. In utero transplantation (IUTx) could correct SCD prior to disease onset, allowing the birth of a healthy baby who requires no treatment and is free from the immense burden this malady exerts. Herein, we provide a brief history of the discovery and characterization of SCD, an overview of current treatment options (highlighting their limitations and risks), and we introduce the concept that treating SCD prior to birth would avoid the hurdles of current treatment options.
Keywords:
clinical management
hematopoietic stem cell (HSC)
HSC transplantation
pathophysiology
sickle cell disease
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