One-Shot gene therapy aims to ease PKU diet burden
NCT ID NCT07318909
First seen Jan 07, 2026 · Last updated May 23, 2026 · Updated 19 times
Summary
This early study tests a single injection of a gene therapy (GS1168) in 9 adults with classic PKU that is not well controlled by diet or other treatments. The goal is to see if it is safe and can lower harmful phenylalanine levels in the blood, possibly allowing a more normal diet. Participants will be followed for one year to check for side effects and changes in their condition.
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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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The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui, 230022, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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