Condition: Metachromatic Leukodystrophy (MLD) · Sponsor: Shenzhen Geno-Immune Medical Institute
This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct gene transfer clinical 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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| Lung-Ji Chang | Shenzhen, Guangdong, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT03725670