Condition: Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID) · Sponsor: Shenzhen Geno-Immune Medical Institute
This is a Phase I/II trial of in vivo lentiviral gene therapy for treating adenosine deaminase severe combined immunodeficiency (ADA-SCID) using a self-inactivating lentiviral vector (LV) ivlv-ADA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous (iv) LV gene therapy protocol.
This description comes directly from the study's public registry record.
Lung-Ji Chang, Ph.D · +86 0755-86573763 · c@szgimi.org
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| Shenzhen Geno-immune Medical Institute | Shenzhen, Guangdong, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT03645460