What is ADA disease?? Please answer!!!?

What is ADA disease? Is it common disease today? Can you name some characteristics?

What does this use for??

Please explain briefly

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  • Anonymous
    1 decade ago
    Favorite Answer

    ADA deficiency is a rare genetic disease. The normal ADA gene

    produces an enzyme called adenosine deaminase that is essential

    for effective immune system function.

    Patients with this condition do not have normal ADA genes, and

    their defective genes do not produce the functional ADA enzyme.

    ADA-deficient children are born with severe immunodeficiency and

    are prone to repeated serious infections, which may be

    life-threatening. Although ADA deficiency can be treated with a

    drug called PEG-ADA, the drug is expensive (more than $60,000 a

    year) and must be taken for life by injection into a vein.

    ADA deficiency was selected for the first approved human gene

    therapy trial for several reasons:

    + The disease is caused by a defect in a single gene, which increases the likelihood that gene therapy will succeed.

    + The gene is regulated in a simple, "always on" fashion,

    unlike many genes whose regulation is complex.

    + The amount of ADA present does not need to be precisely

    regulated. Even small amounts of the enzyme are known to be

    beneficial, while larger amounts are also tolerated well.

    The first clinical trial of gene therapy began in September 1990.

    Two children diagnosed with ADA deficiency were treated with this

    new approach. As a precaution, they also continued to receive

    weekly doses of the drug PEG-ADA. The children's immune status

    improved after they received the gene therapy; however, it worked

    for only a few months and had to be repeated several times over

    the next 2 to 3 years. Since then, the children have had periodic

    tests which confirm that their re-engineered cells are surviving

    and producing the ADA enzyme. Both now take smaller doses of

    PEG-ADA to keep their disease under control.

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