Transamniotic Fetal Delivery of Cystic Fibrosis ( <scp>CF</scp> ) Transmembrane Conductance Regulator ( <scp>CFTR</scp> ) <scp>mRNA</scp>
< 0.001 for all), increasing initially over time. Human CFTR was detected comparably in the lungs from both groups, suggesting interspecies homology at that anatomical site. No human CFTR was detected in maternal serum. Encapsulated exogenous mRNA encoding for the cystic fibrosis transmembrane conductance regulator protein can be incorporated and translated by fetal small bowel cells after simple intra-amniotic injection in a healthy rodent model. Transamniotic mRNA delivery could become a novel strategy for the perinatal management of meconium ileus associated with cystic fibrosis.
Authors
- Dario O. Fauza (ORCID: https://orcid.org/0000-0002-0679-0125)
- Kamila Moskowitzova (ORCID: https://orcid.org/0000-0001-8488-0882)
- D. Zurakowski
- Ashlyn E. Whitlock (ORCID: https://orcid.org/0000-0002-3090-4022)
- Yash V. Shroff
Institutions
- Boston Children's Hospital (US)
- Harvard University (US)
Publication Details
- Journal
- FASEB BioAdvances
- Published
- 2026-04-01
- DOI
- https://doi.org/10.1096/fba.2025-00288
- Primary Topic
- Cystic Fibrosis Research Advances
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Boston Children's Hospital