Single gene shot could tame rare enzyme disorder
NCT ID NCT06818838
First seen Jan 23, 2026 · Last updated Apr 23, 2026 · Updated 10 times
Summary
This early-phase study tests a single-dose gene therapy called LY-M001 in 12 adults with type 1 Gaucher disease, a genetic condition that causes organ enlargement and bone problems. The therapy uses a harmless virus to deliver a working copy of the GBA1 gene to the liver, so the body can produce the missing enzyme. The main goals are to check safety and see if the treatment can improve enzyme levels and reduce liver and spleen size.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Guangzhou First People's Hospital
RECRUITINGGuangzhou, Guangdong, 510000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Hematology Hospital, Chinese Academy of Medical Sciences
RECRUITINGTianjin, Tianjin Municipality, 300011, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Shanxi Bethune Hospital
RECRUITINGTaiyuan, Shanxi, 030000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Conditions
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