Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID) Clinical Trial
Official title:
Gene Transfer for Adenosine Deaminase-severe Combined Immunodeficiency (ADA-SCID) Using an Improved Self-inactivating Lentiviral Vector (TYF-ADA)
Gene transfer for ADA-SCID using an improved lentiviral vector (TYF-ADA)
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. 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. Patients who lack this enzyme are vulnerable to
frequent and severe infections.
ADA-SCID patients are normally rescued by a bone marrow transplant (BMT) from a matched
healthy donor. However, matched donors are difficult to find and donor BMT is associated with
high risk. 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. 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. We will assess the lentiviral gene integration sites and the long-term effect of
this gene transfer procedure.
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