Creating gene-corrected stem cells for Alpha-1 Antitrypsin Deficiency
Part of Digestive system, Genetic & congenital, Lungs & breathing clinical trials.
This trial uses your own cells to create stem cells (iPSCs) that can be genetically corrected to treat severe Alpha-1 Antitrypsin Deficiency. It is an early study exploring a potential cure.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 years or older.
- You must have a confirmed diagnosis of severe Alpha-1 Antitrypsin Deficiency with two specific genetic changes (pathological variants).
- You must not be currently receiving augmentation therapy (infusions of AAT protein).
- You must be willing and able to give informed consent.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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