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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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
Muzumdar, M. D., Tasic, B., Miyamichi, K., Li, L., & Luo, L. (2007) · genesis · doi:10.1002/dvg.20335 · Cited by 3,491
Kosicki, M., Tomberg, K., & Bradley, A. (2018) · Nature Biotechnology · doi:10.1038/nbt.4192 · Cited by 1,883
Haeussler, M., Schönig, K., Eckert, H., Eschstruth, A., Mianné, J., Renaud, J.-B., Schneider-Maunoury, S., Shkumatava, A., Teboul, L., Kent, J., et al. (2016) · Genome biology · doi:10.1186/s13059-016-1012-2 · Cited by 1,756
Rowe, S. M., Miller, S., & Sorscher, E. J. (2005) · New England Journal of Medicine · doi:10.1056/nejmra043184 · Cited by 1,398
Labiris, N., & Dolovich, M. (2003) · British Journal of Clinical Pharmacology · doi:10.1046/j.1365-2125.2003.01892.x · Cited by 1,212
Cas9 as a versatile tool for engineering biology
Mali, P., Esvelt, K. M., & Church, G. M. (2013) · Nature Methods · doi:10.1038/nmeth.2649 · Cited by 1,197
Cited by
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The delivery challenge: fulfilling the promise of therapeutic genome editing
van Haasteren, J., Li, J., Scheideler, O. J., Murthy, N., & Schaffer, D. V. (2020) · Nature Biotechnology · doi:10.1038/s41587-020-0565-5 · Cited by 285
Ionizable lipid nanoparticles for in utero mRNA delivery
Riley, R. S., Kashyap, M. V., Billingsley, M. M., White, B., Alameh, M.-G., Bose, S. K., Zoltick, P. W., Li, H., Zhang, R., Cheng, A. Y., et al. (2021) · Science Advances · doi:10.1126/sciadv.aba1028 · Cited by 222
Self-citationLung and liver editing by lipid nanoparticle delivery of a stable CRISPR–Cas9 ribonucleoprotein
Chen, K., Han, H., Zhao, S., Xu, B., Yin, B., Lawanprasert, A., Trinidad, M. I., Burgstone, B. W., Murthy, N., & Doudna, J. A. (2024) · Nature Biotechnology · doi:10.1038/s41587-024-02437-3 · Cited by 201
Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics
Liu, W., Li, L., Jiang, J., Wu, M., & Lin, P. (2021) · Precision Clinical Medicine · doi:10.1093/pcmedi/pbab014 · Cited by 196
Rasul, M. F., Hussen, B. M., Salihi, A. B., Ismael, B. S., Jalal, P. J., Zanichelli, A., Jamali, E., Baniahmad, A., Ghafouri‐Fard, S., Basiri, A., et al. (2022) · Molecular Cancer · doi:10.1186/s12943-021-01487-4 · Cited by 187
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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