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Experimental gene therapy aims to protect lungs in Alpha-1 patients

NCT ID NCT06996756

First seen Oct 31, 2025 · Last updated May 22, 2026 · Updated 31 times

Summary

This early-stage study tests a one-time gene therapy for alpha-1 antitrypsin deficiency, a genetic condition that can lead to lung damage. Researchers want to see if the treatment is safe and can raise protective protein levels in the blood. The study involves 16 adults with mild to moderate emphysema caused by this deficiency.

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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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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • WCMC Department of Genetic Medicine

    RECRUITING

    New York, New York, 10021, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.