The DOI matches its Crossref record; 35 of the 36 references it cites were found in a registry or an index.
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
Open accessAbstract
Many genetic liver diseases in newborns cause repeated, often lethal, metabolic crises. Gene therapy using nonintegrating viruses such as adeno-associated virus (AAV) is not optimal in this setting because the nonintegrating genome is lost as developing hepatocytes proliferate. We reasoned that newborn liver may be an ideal setting for AAV-mediated gene correction using CRISPR-Cas9. Here we intravenously infuse two AAVs, one expressing Cas9 and the other expressing a guide RNA and the donor DNA, into newborn mice with a partial deficiency in the urea cycle disorder enzyme, ornithine transcarbamylase (OTC). This resulted in reversion of the mutation in 10% (6.7-20.1%) of hepatocytes and increased survival in mice challenged with a high-protein diet, which exacerbates disease. Gene correction in adult OTC-deficient mice was lower and accompanied by larger deletions that ablated residual expression from the endogenous OTC gene, leading to diminished protein tolerance and lethal hyperammonemia on a chow diet.
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“As a result, in applications calling for corrective gene insertion, a second AAV vector encoding the sgRNA and/or a template sequence for homologydirected DNA repair must be used, reducing efficiency due to the need for cells to acquire both AAV vectors at once 59,60 .”
From The promise and challenge of therapeutic genome editing (2020) · cites this paper as [59]
Experimental — accuracy not yet measuredIn the citing paper
“Currently, researchers most frequently employ a dual AAV system for CRISPR-mediated gene correction: one AAV expresses Cas9 while the other expresses the sgRNA and donor template DNA [24].”
From Viral Delivery Systems for CRISPR (2019) · cites this paper as [24]
In the citing paper
“Furthermore, there was an increase in survival of mice with a high-protein diet, a stressor which exacerbates this in-born error of metabolism.”
From Viral Delivery Systems for CRISPR (2019) · cites this paper as [24]
In the citing paper
“After AAV delivery, these mice experienced a reversion of the mutation in 10% of their hepatocytes.”
From Viral Delivery Systems for CRISPR (2019) · cites this paper as [24]
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Cites
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1 of the 36 references couldn’t be matched to a known paper.
Research works printed but not linked: 1
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Controlled linking of AAV capsids enables coordinated multi-vector deliveryPreprint
Kim, Y., Ding, X., Yang, C., Lee, G., Gradinaru, V., & Park, J. (2026) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.64898/2026.04.13.716639
Gene therapy for liver diseases: methods, challenges and opportunities
Yan, M., & Xiang, Q. (2026) · Journal of Nanobiotechnology · doi:10.1186/s12951-026-04518-8
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Karki, P., & Sudarshan, G. C. (2026) · Genetics. · doi:10.5772/intechopen.1015771 · Cited by 1
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Steiman, S., Kinsella, C., Zhang, Y., Omer, S., Brown, C., Forguson, G., Elbakr, L., Pastore, S., & Ivakine, E. A. (2026) · International Journal of Molecular Sciences · doi:10.3390/ijms27156954
“Due to the size of our current CRISPRa strategy with dCas9 and combinatorial guides, delivery using an AAV-based method would require a dual AAV system splitting up the dCas9 and the sgRNAs to overcome the packaging limits [51].”
Martinez, M. A., & Harding, C. O. (2026) · Molecular Therapy · doi:10.1016/j.ymthe.2026.08.015
Gene Therapy Approaches for the Treatment of Phenylketonuria
Martinez, M. A., Young, L. J., Harding, C. O., Schwank, G., & Thöny, B. (2026) · Springer eBooks · doi:10.1007/978-3-032-26690-3_20
A brief history of gene therapy for ornithine transcarbamylase deficiency
Breilyn, M. S., Konczal, L. L., & Harding, C. O. (2026) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2026.110257
Genome Editing for Glycogen Storage Diseases
von Beck, T. A., Wang, R., & Koeberl, D. D. (2026) · Journal of Inherited Metabolic Disease · doi:10.1002/jimd.70249
“Genome editing has been initiated to correct a mutation or integrate a transgene as a method to stably treat liver metabolic diseases, including GSD Ia [25, 62].”
Jia, D., Liu, X., Wang, D., He, X., Liang, X., & Peng, W. (2026) · Journal of Fungi · doi:10.3390/jof12090705
“Second, consistent with findings in most model systems, introducing PAM-disrupting mutations into ssODN templates dramatically elevated HDR efficiency from 7.4% to 58.5%, validating that abolishing repeated Cas-mediated cleavage of repaired loci is an effective optimization approach [24,25].”
InfluentialCRISPR/Cas based genome editing for liver diseases: Promise and challenge
Guan, J., Yang, M., Yang, X., He, Q., Wang, B., & Zhan, C. (2026) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2026.09.027
Metadata
- Authors
- Yang, Y., Wang, L., Bell, P., McMenamin, D., He, Z., White, J., Yu, H., Xu, C., Morizono, H., Musunuru, K., Batshaw, M. L., & Wilson, J. M.
- Affiliations
- Sichuan University (CN)
- University of Pennsylvania (US)
- Children's National (US)
- Brigham and Women's Hospital (US)
- Corresponding author
- Wilson, James M
- ORCID
- Venue
- Nature Biotechnology
- Published
- 2016-02-01
- Volume
- 34
- Issue
- 3
- Pages
- 334-338
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- PMC
- PMC4786489
- OpenAlex
- W2253584908
- Cited by
- 556
- References
- 36
- Citations by year
- 2026: 20 · 2025: 25 · 2024: 36 · 2023: 52 · 2022: 55 · 2021: 61 · 2020: 70 · 2019: 62 · 2018: 72 · 2017: 71 · 2016: 30
- FWCI
- 63.21
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Digital Access to Scholarship at Harvard (DASH) (Harvard University) (submittedVersion, cc-by)
- Funders
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (P01HD057247)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- adeno-associated virus, CRISPR/Cas9, gene correction, ornithine transcarbamylase deficiency, urea cycle disorders, neonatal mice, liver disease, hyperammonemia, high-protein diet, hepatocytes, gene therapy
- MeSH terms
- Dependovirus, Animals, Disease Models, Animal, Genetic Vectors, Humans, Ornithine Carbamoyltransferase, Viruses, RNA Editing, Gene Transfer Techniques, Ornithine Carbamoyltransferase Deficiency Disease, Mice, CRISPR-Cas Systems
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