The DOI matches its Crossref record; 53 of the 53 references it cites were found in a registry or an index.
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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Citations
Cites
Multiplex Genome Engineering Using CRISPR/Cas Systems
Cong, L., Ran, F. A., Cox, D., Lin, S., Barretto, R., Habib, N., Hsu, P. D., Wu, X., Jiang, W., Marraffini, L. A., et al. (2013) · Science · doi:10.1126/science.1231143 · Cited by 15,339
Genome engineering using the CRISPR-Cas9 system
Ran, F. A., Hsu, P. D., Wright, J., Agarwala, V., Scott, D. A., & Zhang, F. (2013) · Nature Protocols · doi:10.1038/nprot.2013.143 · Cited by 11,133
The new frontier of genome engineering with CRISPR-Cas9
Doudna, J. A., & Charpentier, E. (2014) · Science · doi:10.1126/science.1258096 · Cited by 7,062
Webb, A. K. (2006) · Cited by 4,369
A global double-fluorescent Cre reporter mouse
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Items 101–121 of 121
Zukünftige intrauterine Therapien
Ochsenbein‐Kölble, N. (2020) · Der Gynäkologe · doi:10.1007/s00129-020-04567-6
Tissue engineering and transplantation in the fetus
Porada, C. D., Atala, A., & Almeida-Porada, G. D. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-818422-6.00022-8
In utero intervention to stem the damage of cystic fibrosis
Dolgin, E. (2020) · Nature · doi:10.1038/d41586-020-02108-8
Di Gioia, S. (2019) · Open Access Journal of Pulmonary & Respiratory Sciences · doi:10.23880/oajprs-16000124
In UteroStem Cell Transplantation, Enzyme Replacement, and Gene Therapy
MacKenzie, T. C., & Schwab, M. E. (2021) · Wiley eBooks · doi:10.1002/9781119676980.ch30
Applications of innovative gene-editing technologies in respiratory diseases
El-Hashash, A. H. K. (2021) · Elsevier eBooks · doi:10.1016/b978-0-12-821206-6.00004-7
Seraj, Z. I., & Hque, S. (2022) · Journal of Bangladesh Academy of Sciences · doi:10.3329/jbas.v45i2.57208
Surfactant Protein B and C (SP-B and SP-C)
Mulugeta, S., & Beers, M. F. (2021) · Elsevier eBooks · doi:10.1016/b978-0-08-102723-3.00167-0
Self-citationCRISPR Gene Editing Successfully Treats Lethal Monogenic Lung Disease in Utero
(2019) · American Journal of Medical Genetics Part A · doi:10.1002/ajmg.a.61213
Filosofia da Edição Genômica: reducionismo e experimentalismo em engenharia genética
Ferreira, L. R. G. (2022) · Digital Library of Theses and Dissertations (Universidade de São Paulo) · doi:10.11606/d.8.2022.tde-28072022-180905
Prenatal Interventions for the Treatment of Congenital Disorders
Singh, K. (2023) · Springer eBooks · doi:10.1007/978-981-19-6008-6_12
Self-citationUsing CRISPR-Cas9 In Human Fetuses to Prevent Trisomy 16 and Trisomy 22 Induced Miscarriages
Qin, E., & Guilz, N. C. (2022) · Journal of Student Research · doi:10.47611/jsrhs.v11i4.3859
Development of Medicinal Products Based on Gene-Editing Technology: Regulatory Practices
Pokrovsky, N. S., Vodyakova, M. A., Melnikova, E. V., & Меркулов, В. А. (2023) · The Bulletin of the Scientific Centre for Expert Evaluation of Medicinal Products Regulatory Research and Medicine Evaluation · doi:10.30895/1991-2919-2023-481
Playing the genetic lottery: an interview with Kiran Musunuru
Musunuru, K. (2023) · Disease Models & Mechanisms · doi:10.1242/dmm.050508
Self-citationClinical and research innovations in childhood interstitial lung disease (chILD)
Wambach, J., & Vece, T. J. (2024) · Pediatric Pulmonology · doi:10.1002/ppul.27025
Aldahhan, R. A., & El-Sheikh, A. A. (2025) · Translational Research in Anatomy · doi:10.1016/j.tria.2025.100411 · Cited by 1
Bain, A. P., & Austin, M. T. (2026) · Advances in Pediatrics · doi:10.1016/j.yapd.2025.12.010
In utero gene editing and precision medicine
Katz, O. L., Kennedy, K., Berger, N. F., Fadl, N., & Peranteau, W. H. (2026) · Seminars in Pediatric Surgery · doi:10.1016/j.sempedsurg.2026.151678
Self-citationRegenerative medicine in pediatric surgery
Peranteau, W. H., & Coppi, P. D. (2026) · Seminars in Pediatric Surgery · doi:10.1016/j.sempedsurg.2026.151679
Self-citationDelivering the blueprint: Advances and challenges in mRNA therapeutics for the respiratory system
Jia, F., Ma, L., & Li, Y. (2026) · International Journal of Pharmaceutics X · doi:10.1016/j.ijpx.2026.100634
Bioengineering strategies for prenatal therapy
Reynolds, E., Farmer, D. L., & Wang, A. (2026) · Nature Reviews Bioengineering · doi:10.1038/s44222-026-00501-y
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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