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Quizlet – Why Might a Patient With ADA-SCID Be a Good Candidate For Somatic Cell Gene Therapy?

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Quizlet – Why Might a Patient With ADA-SCID Be a Good Candidate For Somatic Cell Gene Therapy?

Why might someone with ADA-scid be an ideal candidate for somatic cell gene therapy?
In X-linked SCID, a single mutated gene causes an enzyme called ADA to be absent or produce very low levels. This prevents lymphocytes responsible for making antibodies from working properly.

Due to this condition, people with severe combined immunodeficiency (SCID) often develop life-threatening infections. Furthermore, those living with ADA-SCID have an increased likelihood of developing cancer or other serious illnesses.

Unfortunately, the only cure for ADA-SCID is a bone marrow transplant (BMT). This involves providing someone with an infusion of healthy blood stem cells from a donor.

Another possible treatment for ADA-SCID is enzyme replacement therapy (ERT), which involves administering injections of healthy enzymes. Although not always successful, ERT may help some individuals with ADA-SCID improve and reduce their chances of contracting an infection.

Another potential cure for ADA-SCID is somatic cell gene therapy, which attempts to correct the gene by inserting a healthy copy into stem cells. This therapy has shown great promise in clinical trials.

It is anticipated that somatic cell gene therapy will become the go-to treatment for ADA-SCID and other genetic diseases. Not only is it more cost-effective than bone marrow transplantation (BMT), but it offers patients living with ADA-SCID an improved quality of life without needing costly antibody infusions or frequent injections of ADA which must be given without a healthy stem cell donor. Despite potential challenges, somatic cell gene therapy has proven safe and successful in curing many children suffering from ADA-SCID.

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