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In utero gene editing for monogenic lung disease
Open accessAbstract
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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“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]
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“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]
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Metadata
- 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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