Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue (Adv. Funct. Mater. 36/2026)
Lipid Nanoparticles In their Research Article (10.1002/adfm.202502540), Steven J. Jonas and co-workers present a lipid nanoparticle (LNP) platform capable of delivering CRISPR/Cas9 machinery configured to achieve site-specific genomic integration of a full length copy of the CFTR gene at its endogenous locus, enabling rescue of the cystic fibrosis disease phenotype. The cover image illustrates LNPs being packaged with gene editing reagents, including mRNA encoding the Cas9 protein, guide RNA, and linear double stranded DNA donor templates used for this mutation agnostic gene correction strategy. The resulting nanoparticles offer a modular platform for the non-viral correction of inherited diseases.
Authors
- Lindsay Lathrop (ORCID: https://orcid.org/0009-0005-1154-9116)
- Priyanka Bhatt (ORCID: https://orcid.org/0000-0002-4531-6952)
- Vrishti Sinha (ORCID: https://orcid.org/0000-0002-2094-387X)
- Donald B. Kohn (ORCID: https://orcid.org/0000-0003-1840-6087)
- Emily Duggan (ORCID: https://orcid.org/0000-0002-6847-7046)
- Brigitte N. Gomperts (ORCID: https://orcid.org/0000-0002-1809-723X)
- Ruth A. Foley (ORCID: https://orcid.org/0000-0002-4271-6045)
- Beate Illek (ORCID: https://orcid.org/0000-0003-0076-3553)
- Alicia Sanoyca (ORCID: https://orcid.org/0009-0004-2238-9683)
- Kevin Coote
- Colin Juett
- Paul G. Ayoub
- Steven J. Jonas
Institutions
- University of San Diego (US)
- University of California, Los Angeles (US)
- Cystic Fibrosis Foundation (US)
- University of California San Diego (US)
- Broad Center (US)
Publication Details
- Journal
- Advanced Functional Materials
- Published
- 2026-05-01
- DOI
- https://doi.org/10.1002/adfm.75501
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 0.00