An LNP-CRISPR gene editing drug demonstrates efficacy and safety in patients with hereditary angioedema following in vivo administration

J Allergy Clin Immunol. 2024 Aug;154(2):272-274. doi: 10.1016/j.jaci.2024.06.011. Epub 2024 Jun 24.

Authors

Norbert Pardi¹, Zoltán Ivics²

Affiliations

¹ Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pa.
² Institute of Clinical Immunology, University of Leipzig, Leipzig, Germany; Fraunhofer Institute for Cell Therapy and Immunology, Leipzig, Germany. Electronic address: [email protected].

PMID: 38925443
DOI: 10.1016/j.jaci.2024.06.011

No abstract available

Keywords: CRISPR/Cas9; Gene editing; gene knockout; hereditary angioedema; in vivo gene delivery; kallikrein; lipid nanoparticle.

Publication types

Letter
Editorial

MeSH terms

Adult
Angioedemas, Hereditary* / drug therapy
Angioedemas, Hereditary* / genetics
CRISPR-Cas Systems
Female
Gene Editing*
Humans
Male
Middle Aged