CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy
Brigham and Women's Hospital · National Institute for Health Research · +1 more institution
Abstract
).
In this phase 1, open-label trial, we administered a single intravenous infusion of nex-z to patients with ATTR-CM. Primary objectives included assessment of the effect of nex-z on safety and pharmacodynamics, including the serum TTR level. Secondary end points included changes in N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels, high-sensitivity cardiac troponin T levels, the 6-minute walk distance, and the New York Heart Association (NYHA) class.
Citation impact
- FWCI
- 40.65
- Percentile
- 100%
- References
- 19
Authors
20- MFMarianna FontanaCorresponding
Brigham and Women's Hospital, National Institute for Health Research, University College London
- SDScott D. Solomon
Brigham and Women's Hospital, National Institute for Health Research, University College London
- JKJessica Kachadourian
Brigham and Women's Hospital, National Institute for Health Research, University College London
- LWLiron Walsh
Brigham and Women's Hospital, National Institute for Health Research, University College London
- RRRicardo Rocha
Brigham and Women's Hospital, National Institute for Health Research, University College London
Topics & keywords
- CRISPR
- Genome editing
- Cardiomyopathy
- Gene
- Genetics
- Biology
- Computational biology
- Medicine
- Zero hunger