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Genetic Approaches to Therapy for the Hemoglobinopathies

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Blood Cell Biochemistry

Part of the book series: Blood Cell Biochemistry ((BLBI,volume 8))

Abstract

Hemoglobinopathies constitute the most common, heterogenous group of genetically inherited disorders. They are caused by an imbalance in the α to β polypeptide ratio (thalassaemia) of hemoglobin (Hb) or abnormalities in the function of the β-globin chain (sickle cell disease) (see Stamatoyannopoulos et al., 1987; Weatherall and Clegg, 1981). It is estimated that approximately 250 million people carry hemoglobinopathies worldwide. This gives rise to more than 300,000 births each year that are homozygous for either thalassaemia or sickle cell disease.

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  1. Department of Experimental Pathology, GKT Medical and Dental School, King’s College, London, Guy’s Hospital, London, SE1 9TR, UK

    Michael Antoniou

  2. Department of Cell Biology, Erasmus University-Rotterdam, 3000DR, Rotterdam, The Netherlands

    Frank Grosveld

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  1. Christie CRC Research Centre Paterson Institute for Cancer Research, Christie Hospital NHS Trust, Manchester, UK

    Leslie J. Fairbairn  & Nydia G. Testa  & 

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Antoniou, M., Grosveld, F. (1999). Genetic Approaches to Therapy for the Hemoglobinopathies. In: Fairbairn, L.J., Testa, N.G. (eds) Blood Cell Biochemistry. Blood Cell Biochemistry, vol 8. Springer, Boston, MA. https://doi.org/10.1007/978-1-4615-4889-8_8

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