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 transduce patient-derived hematopoietic stem cells (HSCs), with the goal of achieving therapeutic gene correction through transplantation of genetically modified HSCs. The primary objectives are to evaluate the safety and efficacy of the gene therapy clinical protocol.
This description comes directly from the study's public registry record.
Lung-Ji Chang, Ph.D · 86-13671121909 · 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/NCT07046338