NTLA-2001: opening a new era for gene therapy

Tongtong Cui, Bojin Li, Wei Li

PDF(179 KB)
PDF(179 KB)
Life Medicine ›› 2022, Vol. 1 ›› Issue (2) : 49-51. DOI: 10.1093/lifemedi/lnac036
News & Opinion
News & Opinion

NTLA-2001: opening a new era for gene therapy

Author information +
History +

Cite this article

Download citation ▾
Tongtong Cui, Bojin Li, Wei Li. NTLA-2001: opening a new era for gene therapy. Life Medicine, 2022, 1(2): 49‒51 https://doi.org/10.1093/lifemedi/lnac036

References

[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.

RIGHTS & PERMISSIONS

2022 The Author(s) 2022. Published by Oxford University Press on behalf of Higher Education Press.
AI Summary AI Mindmap
PDF(179 KB)

Accesses

Citations

Detail

Sections
Recommended

/