Gene Therapy for “Bubble Boy” Disease
نویسنده
چکیده
Adenosine deaminase (ADA) deficiency results in the accumulation of toxic metabolites that destroy the immune system, causing severe combined immunodeficiency (ADA-SCID), often referred to as the "bubble boy" disease. Strimvelis is a European Medicines Agency approved gene therapy for ADA-SCID patients without a suitable bone marrow donor.
منابع مشابه
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ورودعنوان ژورنال:
- Cell
دوره 166 شماره
صفحات -
تاریخ انتشار 2016