The role of OMICS research in understanding phenotype variation in thalassaemia: the THALAMOSS project

@inproceedings{Gambari2014TheRO,
  title={The role of OMICS research in understanding phenotype variation in thalassaemia: the THALAMOSS project},
  author={Roberto Gambari},
  year={2014}
}
The β-thalassaemias are a group of severe and rare anaemias with monogenic inheritance, a complex systemic phenotype and several treatment-related complications, caused by more than 300 mutations of the β-globin gene. Novel therapeutic protocols, most of which are based on still experimental treatments, show great promise but significant variability of success between patients. These strategies include chemical/ molecular induction of the endogenous β-like g-globin gene or the restoration of… CONTINUE READING

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