What are the two methods for correcting ADA deficiency in a child?
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Gene therapy is the process of introduction of DNA into an organism e.g. human beings in order to treat a disease. It is used to replace a missing gene product or to correct mutant alleles. ADA is an autosomal-recessive inherited disorder that occurs due to defective adenosine deaminase (ADA) enzyme.
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The most successful treatment for ADA deficiency is histocompatible bone marrow transplantation from an HLA-matched sibling. Because this treatment option is seldom available, alternative treatments have been identified, including T cell–depleted haploidentical bone marrow transplantation from a parent.
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