Strimvelis Gene Therapy for ADA-SCID Connected to Leukemia Case

  While gene therapy offers the promise of addressing and treating many genetic disorders, creating a better quality of life for many people, there are also ethical issues associated with…

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Follow-up from ADA-SCID Trial Shows Impressive Results Compared to Historical Data

ADA-SCID ADA-SCID is a form of Severe Combined Immunodeficiency (SCID) which is caused by Adenosine Deaminase Deficiency. A lack of the ADA gene in the body results in impaired development of the…

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Why Gene Therapy Can be a Boom or Bust for ADA Patients
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Why Gene Therapy Can be a Boom or Bust for ADA Patients

Have you ever heard of “ex vivo” gene therapy? If you haven't, it means removing some cells, modifying them, and putting them back in. The goal? Curing the diseases that…

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