Gene therapy for the haemoglobinopathies

Hemoglobinopatiler gen terapi protokollerinin geliştirildiği ilk hastalıklardandır. İlk denemeler $beta$ -globin geninin bilinen hücre hatları ve daha sonrasında fare modellerde viral vektörlerle transfer edilmesi üzerine gerçekleşti. Bu denemeler başarıya ulaşamadı. Çünkü viral vektörler kullanılan sistemlerde dezavantajlara sahipti. Aynı zamanda $beta$ -globin geni oldukça kompleks bir ekspresyona sahip olduğu için başarılı olunamadı. Bugün daha fazla umut vadeden vektörler, viral veya episomik olup, daha büyük DNA parçalarının aktarılması ve transgenlerin uzun dönemli ekspresyonlarının desteklenerek insan kök hücrelerinin uyarılmasını sağlıyorlar.

Hemoglobinopati için gen terapisi

The haemoglobinopathies have been among the first diseases to be considered for the development of gene therapy protocols. Initial attempts involved the transfer of the $beta$ -globin gene into established cell lines and later mouse models of the disease, by means of viral vectors. These attempts were not successful, due to various disadvantages of the vector system used, as well as to the complex regulation of $beta$ -globin gene expression. More promising are current attempts that make use of vectors, viral or episomic, that can accommodate large insert DNA, can support long term expression of the transgenes and can transduce human haemopoetic stem cells.

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