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Folia Medica Indonesiana

ISSN 0303-7932

Vol. 34 / No. 4 / Published : 1998-10

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Original Article :

The prospect of gene therapy for thalassemic patients, a review

Author :

      Abstract :

      Gene therapy is the genetic modification of the cells of a patient to combat disease. This very broad definition includes many different possible approaches and can involve transfer of cloned human genes, double stranded human gene segments, genes from other genomes, oligonucleotides and various artificial genes, such as antisense genes. Such gene transfer can result in genetic modification of the cells of the patient. Although recessively inherited disorders are, in principle, amenable to gene augmentation therapy, certain disorders are less amenable than others. A good example is provided by  thalassemia, which results from mutations in the  globin gene. This is a severe disorder affecting hundreds or thousands of people worldwide, especially in Southeast Asia, and superficially would appear to be an excellent candidate for gene therapy. The gene is very small and has been characterized extensively, the disorder is recessively inherited and affects blood cells, but there are problems still to be solved, these are: the inefficient gene transfer, the poor expression and the very tight control of gene expression required following insertion of a normal  globin gene into the desired cells. Furthermore, the amount of  globin product must be made equal to the amount of  globin.

      Keyword :

      thalassemia, gene therapy, methods, vectors, retrovirus, gene expression,

      References :


        Archive Article

        Cover Media Content

        Volume : 34 / No. : 4 / Pub. : 1998-10
        1. The Prospect Of Gene Therapy For Thalassemic Patients, A Review
        2. Growth In Indonesian Children Aged 6 To 15 Years
        3. Pharmacokinetics Of Methotrexate In Children
        4. Echocardiography Follow-up After Succesful Percutaneous Transvenous Mitral Commisurotomy (three Years Follow-up)
        5. Catheter Rupture During Angiography (a Case Report)
        6. Occupational Environment And Male Reproductive Health
        7. Prescribing For Cough: The Use Of Antitussive And Expectorants
        8. Jensen Procedure On Paralytic Esotropia Or Lateral Rectus Or N Vi Palsy Caused By Trauma
        9. A Preliminary Report, Staining And Visualization Of Dna In Agarose-gel With Bio-safe Dye
        10. The Use Of Unit Cost Analysis In Health Center Management, A Study In Lawang Health Center