![]() We will assess the lentiviral gene integration sites and the long-term effect of this gene transfer procedure. By collecting an individual's stem cells and modifying them with a lentivirus, the gene-corrected cells can be returned to the patient to help produce normal healthy immune cells.The primary objectives are to evaluate the safety of the improved self-inactivating lentiviral vector TYF-ADA, the ex vivo gene transfer clinical protocol and the efficacy of immune reconstitution in patients overcoming frequent infections present at the time of treatment. #SCID ADENOSINE DEAMINASE TRIAL#This trial aims to treat ADA-SCID using a safety and efficiency improved self-inactivating lentiviral vector carrying a functional ADA gene to correct the genetic defect. However, matched donors are difficult to find and donor BMT is associated with high risk. Patients who lack this enzyme are vulnerable to frequent and severe infections.ĪDA-SCID patients are normally rescued by a bone marrow transplant (BMT) from a matched healthy donor. Those are the medical termsbut what do they actually mean Here’s what goes on inside the body of someone with ADA-SCID: Every day, cells divide in your body. ADA stands for adenosine deaminase, and SCID stands for severe combined immune deficiency. Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). This gene encodes for the adenosine deaminase enzyme, which is essential for the proper growth and function of infection-fighting white blood cells called T and B lymphocytes. People with ADA-SCID have a deficiency in the ADA enzyme. This clinical trial will evaluate a safety and efficiency improved lentiviral vector system for delivering a therapeutic gene to patients with severe combined immunodeficiency (SCID) due to a defective adenosine deaminase (ADA) gene. Why Should I Register and Submit Results? Adenosine deaminase ( ADA) gene defects are among the most common causes of SCID. ![]()
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