Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting <i>ANGPTL3</i>
Editing of <i>ANGPTL3</i> was associated with few adverse events and resulted in reductions from baseline in ANGPTL3 levels. (Funded by CRISPR Therapeutics; Australia New Zealand Clinical Trials Registry number, ACTRN12623000809639.).
Authors
- Jen Nielsen
- John E. Baker (ORCID: https://orcid.org/0000-0001-7899-3266)
- Steven E. Nissen (ORCID: https://orcid.org/0000-0002-7231-6464)
- Jason M. Duran (ORCID: https://orcid.org/0000-0003-1666-2319)
- Ashish Sarraju (ORCID: https://orcid.org/0000-0003-1649-2110)
- Huansheng Xu
- Stephen James Nicholls (ORCID: https://orcid.org/0000-0002-9668-4368)
- Nipam H. Patel (ORCID: https://orcid.org/0000-0003-4328-654X)
- Qiuqing Wang
- Luke J. Laffin (ORCID: https://orcid.org/0000-0002-3262-792X)
- Russell Scott (ORCID: https://orcid.org/0000-0002-2107-6480)
- Peter Clifton (ORCID: https://orcid.org/0000-0002-6411-626X)
- Shweta Singh (ORCID: https://orcid.org/0009-0006-0372-3628)
- Kathy Wolski
Institutions
- Cleveland Clinic (US)
- Royal Adelaide Hospital (AU)
- Clinical Trials New Zealand (NZ)
- CRISPR Therapeutics (Switzerland) (CH)
- Center for Clinical Research (United States) (US)
- Monash University (AU)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2025-11-08
- DOI
- https://doi.org/10.1056/nejmoa2511778
- Citations
- 28
- Primary Topic
- Lipid metabolism and disorders
- Type
- article
- Field-Weighted Citation Impact
- 38.87