First-in-Human gene therapy trial launches for devastating childhood disease
NCT ID NCT07398872
Summary
This is an early-stage study to see if a new gene therapy called AAV9.hMCOLN1co is safe for children with Mucolipidosis Type IV (MLIV), a rare and serious genetic disorder. The therapy aims to deliver a working copy of the faulty gene to try to control the disease. The study will enroll a small number of children, aged 1.5 to 8 years, to closely monitor their health after receiving the treatment.
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Contacts and locations
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Locations
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Children's Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Conditions
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