Abstract
Sickle cell disease (SCD) is an inherited hemoglobinopathy that affects millions of people worldwide with increasing prevalence. SCD results in a substantial burden with progressive organ damage and recurrent painful crises (vaso-occlusive crises; VOCs). While hematopoietic stem cell transplantation and gene therapy are emerging options, they are not accessible to most patients
| Original language | English |
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| Qualification | Doctor of Philosophy |
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| Supervisors/Advisors |
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| Award date | 19 Sept 2024 |
| Print ISBNs | 9789465061313 |
| Publication status | Published - 2024 |
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