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Can haemophilia be cured by gene therapy?

Q: What are latest developments to cure Haemophilia-A (Factor VIII deficient) particularly in gene therapy?

A:Haemophilia, theoretically, is eminently suited for gene therapy as the patients have a single defective gene, which can be substituted by a normal gene and the gene product is required in small amounts for normal physiological functioning. If the normal gene can be directed to the proper cells and in adequate amount, the clotting factor so produced will restore the clotting mechanism. Despite gene therapy, the genetic defect will continue to be passed to the offspring as this modality can only correct the defect and not the genetic line. The three main problems which still remain are finding the right vector, getting into the right cells and avoiding rejection by the body. A technique called chimeroplasty is being tried in animal models where attempt has been made to alter the genetic code. If successful, this would truly cure the disease. You can also refer to the following website for more information: http://www.therapeuticcloning.ca/index.php?C=Gene%20Therapy&T=gene-therapy&D=gene-hemophilia-therapy


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