what is a single gene disorder in this evidence

Question

The phase 1 trial sponsored by Intellia Therapeutics and Regeneron Pharmaceuticals showed that lipid nanoparticles can effectively deliver genome-editing treatment systemically, leading to reductions in TTR protein levels in patient volunteers, with higher dosages resulting in greater reductions.

Answer 1

The evidence suggests that the single gene disorder being targeted in this trial is transthyretin amyloidosis (ATTR), which is characterized by high levels of the TTR protein. The genome-editing treatment is designed to reduce TTR protein levels, indicating that this disorder is caused by a mutation in a single gene (TTR gene).