NTLA-2001: opening a new era for gene therapy
Tongtong Cui, Bojin Li, Wei Li
NTLA-2001: opening a new era for gene therapy
[1] |
GillmoreJD, GaneE, TaubelJ, et al. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis. N Engl J Med 2021;385:493–502.
CrossRef
Google scholar
|
[2] |
AimoA, Castiglione V, RapezziC, et al. RNA-targeting and gene editing therapies for transthyretin amyloidosis. Nat Rev Cardiol 2022;19:655–67.
CrossRef
Google scholar
|
[3] |
AdliM. The CRISPR tool kit for genome editing and beyond. Nat Commun 2018;9:1–13.
CrossRef
Google scholar
|
[4] |
FinnJD, SmithAR, PatelMC, et al. A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing. Cell Rep 2018;22:2227–35.
CrossRef
Google scholar
|
[5] |
WoodK, PinkM, SeitzerJ, et al. Development of NTLA-2001, a CRISPR/Cas9 genome editing therapeutic for the treatment of ATTR. Presented at the Second European Congress for ATTR Amyloidosis, Berlin, September 1–3, 2019.
|
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