Question
what is a single gene disorder in this evidence
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.
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.
Answers
GPT 3.5
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).