ArticleScientific reports2017
Proof-of-Concept Gene Editing for the Murine Model of Inducible Arginase-1 Deficiency.
Article in Scientific reports, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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Who cites it
10 citing papers in PubMed, 19 citations in OpenAlex.
- Application of Induced Pluripotent Stem Cells (iPSCs) in Hereditary and Viral Diseases of the Liver: Modeling and Treatment.International journal of molecular sciences · 2025Review
- Precision in Action: The Role of Clustered Regularly Interspaced Short Palindromic Repeats/Cas in Gene Therapies.Vaccines · 2024Review
- Review: Research progress of adipose-derived stem cells in the treatment of chronic wounds.Frontiers in chemistry · 2023Review
- Modelling urea cycle disorders using iPSCs.NPJ Regenerative medicine · 2022Review
- Review
- Review
- Genome editing technologies to treat rare liver diseases.Translational gastroenterology and hepatology · 2020Review
- Novel Approaches for the Treatment of Familial Hypercholesterolemia: Current Status and Future Challenges.Journal of atherosclerosis and thrombosis · 2018Review
- Transplantation of Gene-Edited Hepatocyte-like Cells Modestly Improves Survival of Arginase-1-Deficient Mice.Molecular therapy. Nucleic acids · 2018Article
- Antitumor activity of EGFR-specific CAR T cells against non-small-cell lung cancer cells in vitro and in mice.Cell death & disease · 2018Article
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
Abstract
Arginase-1 deficiency in humans is a rare genetic disorder of metabolism resulting from a loss of arginase-1, leading to impaired ureagenesis, hyperargininemia and neurological deficits. Previously, we generated a tamoxifen-inducible arginase-1 deficient mouse model harboring a deletion of Arg1 exons 7 and 8 that leads to similar biochemical defects, along with a wasting phenotype and death within two weeks. Here, we report a strategy utilizing the Clustered, Regularly Interspaced, Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system in conjunction with piggyBac technology to target and reincorporate exons 7 and 8 at the specific Arg1 locus in attempts to restore the function of arginase-1 in induced pluripotent stem cell (iPSC)-derived hepatocyte-like cells (iHLCs) and macrophages in vitro. While successful gene targeted repair was achieved, minimal urea cycle function was observed in the targeted iHLCs compared to adult hepatocytes likely due to inadequate maturation of the cells. On the other hand, iPSC-derived macrophages expressed substantial amounts of "repaired" arginase. Our studies provide proof-of-concept for gene-editing at the Arg1 locus and highlight the challenges that lie ahead to restore sufficient liver-based urea cycle function in patients with urea cycle disorders.
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