In utero gene editing for monogenic lung disease
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Prenatal CRISPR gene editing results in efficient pulmonary epithelial cell editing and ameliorates a mouse model of a congenital lung disease.
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In utero gene editing has rescued the lethal phenotype of hereditary tyrosinemia type 1, improved the pulmonary phenotype of surfactant protein C deficiency, and ameliorated metabolic, musculoskeletal, and cardiac disease in mucopolysaccharidosis type I (MPS-IH, Hurler syndrome) in mouse models.
“Previously, we demonstrated the therapeutic potential of in utero gene editing in mouse models of human diseases, rescuing the lethal phenotype of hereditary tyrosinemia type 1, improving the pulmonary phenotype of surfactant protein C deficiency, and ameliorating metabolic, musculoskeletal, and cardiac disease in mucopolysaccharidosis type I (MPS-IH, Hurler syndrome) (7–9).”
From Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease (2023) · cites this paper as [8]
CNS targeting via prior viral vector approaches was not efficient.
“Although encouraging, CNS targeting via these approaches was not efficient, highlighting the need to evaluate alternative delivery approaches to treat genetic CNS diseases.”
From Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease (2023) · cites this paper as [8]
The Introduction and Discussion describe viral-vector delivery and gene editing targeted to fetal lung cells, not the CNS. Searches for CNS targeting, brain transduction, and inefficient viral-vector delivery found no passage in our copy assessing the efficiency of prior approaches for CNS targeting. Page numbers were not available. (LLM, conf=0.77)
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- Authors
- Alapati, D., Zacharias, W. J., Hartman, H. A., Rossidis, A. C., Stratigis, J. D., Ahn, N. J., Coons, B., Zhou, S., Li, H., Singh, K., Katzen, J., Tomer, Y., Chadwick, A. C., Musunuru, K., Beers, M. F., Morrisey, E. E., & Peranteau, W. H.
- Affiliations
- Alfred I. duPont Hospital for Children (US)
- Cincinnati Children's Hospital Medical Center (US)
- Children's Hospital of Philadelphia (US)
- University of Pennsylvania (US)
- Corresponding author
- Morrisey, Edward E.
- Peranteau, William H.
- ORCID
- Venue
- Science Translational Medicine
- Published
- 2019-04-17
- Volume
- 11
- Issue
- 488
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- PMC
- PMC6822403
- OpenAlex
- W2937051914
- Cited by
- 123
- References
- 53
- Citations by year
- 2026: 10 · 2025: 13 · 2024: 13 · 2023: 20 · 2022: 17 · 2021: 25 · 2020: 18 · 2019: 6
- FWCI
- 5.46
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- VUBIR (Vrije Universiteit Brussel) (submittedVersion)
- Funders
- NIH Office of the Director (HL134745, HL132999, HL132349, HL007843, HL118744, HL126875, TR001878, T32HL007586, HL007586, HL119436)
- National Center for Advancing Translational Sciences (TL1TR001880)
- U.S. Department of Veterans Affairs (1I01BX001176)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Neonatal Respiratory Health Research
- Keywords
- fetal gene therapy, lung morphology, survival, postnatal mice
- MeSH terms
- Gene Editing, Animals, Disease Models, Animal, Epithelial Cells, Humans, Lung Diseases, Mutation, Pulmonary Surfactant-Associated Protein C, Mice, CRISPR-Cas Systems
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