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Julian David Gillmore, Ed J. Gane, Jörg Täubel, Justin Kao, Marianna Fontana, Michael L. Maitland, Jessica L. Seitzer, Daniel O’Connell, Kathryn Rose Walsh, Kristy M. Wood and 16 more
BACKGROUND: . METHODS: After conducting preclinical in vitro and in vivo studies, we evaluated the safety and pharmacodynamic effects of single escalating doses of NTLA-2001 in six patients with hereditary ATTR amyloidosis with polyneuropathy, three in each of the two initial dose groups (0.1 mg per kilogram and 0.3 mg per kilogram), within an ongoing phase 1 clinical study. RESULTS: after a single dose. Serial assessments of safety during the first 28 days after infusion in patients revealed few adverse events, and those that did occur were mild in grade. Dose-dependent pharmacodynamic effects were observed. At day 28, the mean reduction from baseline in serum TTR protein concentration was 52% (range, 47 to 56) in the group that received a dose of 0.1 mg per kilogram and was 87% (range, 80 to 96) in the group that received a dose of 0.3 mg per kilogram. CONCLUSIONS: . (Funded by Intellia Therapeutics and Regeneron Pharmaceuticals; ClinicalTrials.gov number, NCT04601051.).
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Gillmore, J. D., Gane, E. J., Täubel, J., Kao, J., Fontana, M., Maitland, M. L., Seitzer, J. L., O’Connell, D., Walsh, K. R., Wood, K. M., Phillips, J. A., Xu, Y., Amaral, A., Boyd, A. P., Cehelsky, J. E., McKee, M. D., Schiermeier, A., Harari, O., Murphy, A., … Lebwohl, D. (2021). CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. New England Journal of Medicine, 385(6), 493–502. https://doi.org/10.1056/nejmoa2107454
Gillmore, Julian David, et al. "CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis." New England Journal of Medicine, vol. 385, no. 6, 2021, pp. 493–502, https://doi.org/10.1056/nejmoa2107454.
Gillmore, Julian David, Ed J. Gane, Jörg Täubel, Justin Kao, Marianna Fontana, Michael L. Maitland, Jessica L. Seitzer, et al. "CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis." New England Journal of Medicine 385, no. 6 (2021): 493–502. https://doi.org/10.1056/nejmoa2107454.
Gillmore J. D., Gane E. J., Täubel J. [и др.] CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis // New England Journal of Medicine. — 2021. — Vol. 385, № 6. — P. 493–502. — URL: https://doi.org/10.1056/nejmoa2107454
@article{gillmore2021crisprcas,
title = {{CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis}},
author = {Julian David Gillmore and Ed J. Gane and Jörg Täubel and Justin Kao and Marianna Fontana and Michael L. Maitland and Jessica L. Seitzer and Daniel O’Connell and Kathryn Rose Walsh and Kristy M. Wood and Jonathan A. Phillips and Yuanxin Xu and Adam Amaral and Adam P. Boyd and Jeffrey E. Cehelsky and Mark D. McKee and Andrew Schiermeier and Olivier Harari and Andrew Murphy and Christos A. Kyratsous and Brian Zambrowicz and Randy Soltys and David E. Gutstein and John P. Leonard and Laura Sepp‐Lorenzino and David Lebwohl},
journal = {New England Journal of Medicine},
year = {2021},
volume = {385},
number = {6},
pages = {493--502},
doi = {10.1056/nejmoa2107454},
url = {https://doi.org/10.1056/nejmoa2107454}
}TY - JOUR AU - Gillmore, Julian David AU - Gane, Ed J. AU - Täubel, Jörg AU - Kao, Justin AU - Fontana, Marianna AU - Maitland, Michael L. AU - Seitzer, Jessica L. AU - O’Connell, Daniel AU - Walsh, Kathryn Rose AU - Wood, Kristy M. AU - Phillips, Jonathan A. AU - Xu, Yuanxin AU - Amaral, Adam AU - Boyd, Adam P. AU - Cehelsky, Jeffrey E. AU - McKee, Mark D. AU - Schiermeier, Andrew AU - Harari, Olivier AU - Murphy, Andrew AU - Kyratsous, Christos A. AU - Zambrowicz, Brian AU - Soltys, Randy AU - Gutstein, David E. AU - Leonard, John P. AU - Sepp‐Lorenzino, Laura AU - Lebwohl, David TI - CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis JO - New England Journal of Medicine VL - 385 IS - 6 SP - 493 EP - 502 PY - 2021 DA - 2021/06/26 DO - 10.1056/nejmoa2107454 UR - https://doi.org/10.1056/nejmoa2107454 ER -
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