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The use of gene therapy to treat genetic diseases

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Hemophilia is a recessive, Xlinked disease; terefore, gnetic segregation and dominance play an important role in the inheritance of hemophilia. Te recessive mutated forms of clotting factor VIII and IX are present on the X chromosome. Terefore, te disease is more common in males because they have a single X chromosome and if it has the mutated gene then the person will have hemophilia as unlike females they do not have another X chromosome for compensation. I females, te dominant X chromosome masks the recessive one; terefore, fmales hemophilia only if both of their X chromosomes have the mutated gene for hemophilia.

Te genetic pattern followed in the inheritance of hemophilia is as follows: Acarrier female and a hemophilic male will bear daughters having an equal chance of being carriers or hemophiliacs, wile the sons have an equal probability of being hemophiliacs or normal. Te experimental technique which employs genes for the treatment and prevention of diseases is referred to as gene therapy. Te exponential progress in the field of gene therapy has enabled doctors to treat by insertion of genes into the patients’ genome.

Te concept of gene therapy revolves around various aspects including the replacement of disease causing mutated genes with normal genes, iactivation of mutated genes and the introduction of new genes into the human body for the treatment of diseases. Cstic fibrosis is one of the most common monogenic autosomal recessive diseases. Te morbidity and mortality associated with cystic fibrosis can be decreased by the replacement of cystic fibrosis transmembrane conductance regulator (CFTR) gene during the neonatal period. Anumber of patients received vivo gene transfers.

Crrently, te key areas of concern in the treatment of cystic fibrosis through gene therapy are choice of vector, mde of delivery to airways, gnetic information translocation and enhancing the expression level of normalized cystic CFTR gene. Crtain viruses behave as vectors and Adenovirus and adeno-associated virus were proposed to be employed in gene therapy for delivering normalized CFTR gene to the airways. Hwever, te use of adeno viruses in humans poses the problem of inducing humoral immunity which is the reason why their has been Te adeno-associated virus does not induce significant inflammation and other adverse effects in both animal and. ..

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preview essay on The use of gene therapy to treat genetic diseases
  • Pages: 4 (1000 words)
  • Document Type: Essay
  • Subject: Biology
  • Level: Undergraduate
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