Gene-Editing breakthrough tested for rare, fatal heart and nerve disease
NCT ID NCT04601051
Summary
This early-stage study tested a new, one-time gene-editing treatment called NTLA-2001 for people with hereditary forms of amyloidosis, a disease where a harmful protein builds up and damages the heart or nerves. The main goal was to check the safety of the treatment and see how it behaves in the body. Researchers also looked for early signs that the treatment could reduce the harmful protein and potentially slow the disease.
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Contacts and locations
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Locations
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Clinical Trial Site
Paris, France
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Clinical Trial Site
Auckland, New Zealand
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Clinical Trial Site
Umeå, Sweden
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Clinical Trial Site
London, United Kingdom
Conditions
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