# CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

> In a small group of patients with hereditary ATTR amyloidosis with polyneuropathy, administration of NTLA-2001 was associated with only mild adverse events and led to decreases in serum TTR protein concentrations through targeted knockout of <i>TTR</i>. (Funded by Intellia Therapeutics and Regeneron...

## Metadata
- Authors: Julian D. Gillmore, Ed Gane, Jörg Täubel, Justin Kao, Marianna Fontana, Michael L. Maitland, Jessica Seitzer, Daniel J. O’Connell, Kathryn Walsh, Kristy Wood, Jonathan A. Phillips, Yuanxin Xu, Adam Amaral, Adam P. Boyd, Jeffrey Cehelsky, Mark D. McKee, Andrew Schiermeier, Olivier Harari, Andrew Murphy, Christos A. Kyratsous, Brian Zambrowicz, Randy Soltys, David E. Gutstein, John P. Leonard, Laura Sepp‐Lorenzino, David Lebwohl
- Journal: New England Journal of Medicine
- Published: 2021-06-26
- DOI: https://doi.org/10.1056/nejmoa2107454
- Citations: 1,572
- Source: OpenAlex
- Access: Open Access

## Technology Hub
- Hub: CRISPR Gene Editing
- Discipline: Biology / Genetics
- Hub URL: https://science-database.com/technology/crispr
- Hub llms.txt: https://science-database.com/technology/crispr/llms.txt

## Abstract
In a small group of patients with hereditary ATTR amyloidosis with polyneuropathy, administration of NTLA-2001 was associated with only mild adverse events and led to decreases in serum TTR protein concentrations through targeted knockout of <i>TTR</i>. (Funded by Intellia Therapeutics and Regeneron Pharmaceuticals; ClinicalTrials.gov number, NCT04601051.).

## Links
- DOI: https://doi.org/10.1056/nejmoa2107454
- OpenAlex: https://openalex.org/W3177445623
- PDF: https://www.nejm.org/doi/pdf/10.1056/NEJMoa2107454?articleTools=true
- JSON API: https://science-database.com/api/v1/technology/crispr

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Paper ID: oa-W3177445623 | Hub: crispr