Modifiers of γ-Globin Gene Expression and Treatment of β-Thalassemia

Beta thalassemia (β-thalassemia) is an autosomal recessive genetic disease with many genes involved. It is a heterogeneous disorder caused by variations in the inactivation mechanism of the Beta-globin (β-globin) genes. Despite seemingly similar genotypes, the patients with Beta-thalassemia have a r...

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Main Authors: Munshi, Anjana (Author), Dadeech, Sneha (Author), Babu, M. Sai (Author), Khetarpal, Preeti (Author)
Format: Ebooks
Published: IntechOpen, 2015-11-11.
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001 intechopen_books_4729
042 |a dc 
100 1 0 |a Munshi, Anjana  |e author 
700 1 0 |a Dadeech, Sneha  |e author 
700 1 0 |a Babu, M. Sai  |e author 
700 1 0 |a Khetarpal, Preeti  |e author 
245 0 0 |a Modifiers of γ-Globin Gene Expression and Treatment of β-Thalassemia 
260 |b IntechOpen,   |c 2015-11-11. 
500 |a https://mts.intechopen.com/articles/show/title/modifiers-of-globin-gene-expression-and-treatment-of-thalassemia 
520 |a Beta thalassemia (β-thalassemia) is an autosomal recessive genetic disease with many genes involved. It is a heterogeneous disorder caused by variations in the inactivation mechanism of the Beta-globin (β-globin) genes. Despite seemingly similar genotypes, the patients with Beta-thalassemia have a remarkable variability in anaemia, growth development, and hepatospleenomegaly and transfusion requirements. The genetic factors may differ in each race or ethnic group for therapy and prevention. Despite remarkable successes in the treatment of Beta-thalassemia in the past decades, it is still the leading cause of death and premature disability in developed and developing countries. Possible factors that influence the severity of anaemia in thalassemia may be inherited or non-inherited. The inherited factors include the type of β-thalassemia, coinheritance of alpha thalassemia (α-thalassemia) and factors that stimulate fetal hemoglobin (HbF) production. In this chapter, respective contributions of known modifiers and also the pharmaceutical agents currently in use and under clinical trials for regulating the globin gene expression will be discussed. 
540 |a https://creativecommons.org/licenses/by/3.0/ 
546 |a en 
690 |a Inherited Hemoglobin Disorders 
655 7 |a Chapter, Part Of Book  |2 local 
786 0 |n https://www.intechopen.com/books/4729 
787 0 |n ISBN:978-953-51-2198-5 
856 \ \ |u https://mts.intechopen.com/articles/show/title/modifiers-of-globin-gene-expression-and-treatment-of-thalassemia  |z Get Online