Ttr-flox 基因敲除小鼠

1. Aimo, Alberto, Castiglione, Vincenzo, Rapezzi, Claudio, Passino, Claudio, Emdin, Michele. 2022. RNA-targeting and gene editing therapies for transthyretin amyloidosis. In Nature reviews. Cardiology, 19, 655-667. doi:10.1038/s41569-022-00683-z. https://pubmed.ncbi.nlm.nih.gov/35322226/

2. Adams, David, Cauquil, Cecile, Labeyrie, Céline, Algalarrondo, Vincent, Théaudin, Marie. 2016. TTR kinetic stabilizers and TTR gene silencing: a new era in therapy for familial amyloidotic polyneuropathies. In Expert opinion on pharmacotherapy, 17, 791-802. doi:10.1517/14656566.2016.1145664. https://pubmed.ncbi.nlm.nih.gov/26800456/

3. Kotit, Susy. 2023. Lessons from the first-in-human in vivo CRISPR/Cas9 editing of the TTR gene by NTLA-2001 trial in patients with transthyretin amyloidosis with cardiomyopathy. In Global cardiology science & practice, 2023, e202304. doi:10.21542/gcsp.2023.4. https://pubmed.ncbi.nlm.nih.gov/37928601/