CRISPR Base Editing for a Monogenic Liver Disorder: Off-Target Assessment and Long-Term Follow-Up Obligations

Authors

  • Csenge Fodorne Department of Cell and Gene Therapy, Orseg Centre for Experimental Therapeutics, Szentgotthard, Hungary Author
  • Torstein Fyksen Department of Clinical Genetics, Salten Centre for Experimental Therapeutics, Fauske, Norway Author
  • Otso Jarvenpaa Centre for Research Ethics, Kanta-Hame Institute of Translational Medicine, Hameenlinna, Finland Author

DOI:

https://doi.org/10.5281/zenodo.22649285

Keywords:

CRISPR Base Editing for a Monogenic Liver Disorder;, Translational medicine; Evidence synthesis, Clinical implementation; Health services research

Abstract

CRISPR Base Editing for a Monogenic Liver Disorder is an area of active development in translational medicine, with evidence
that has accumulated unevenly across its component questions. We review the current evidence base for crispr base editing for a
monogenic liver disorder, spanning analytical, clinical, implementation, governance, and economic dimensions, focusing on
where practice has converged and where it continues to vary, and set out the practical considerations relevant to a service
considering its adoption. We conclude that selective, context-specific use is best supported by the evidence currently available,
rather than uniform adoption or avoidance.

References

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Alvez D, Dumitrescu A, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a systematic review". BMJ Open 28(2011): 410-419. DOI: 10.3390/diagnostics15162117

Brankovic K, Kowalczyk B, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a methodological appraisal". J Clin Epidemiol 30(2014): 421-431. DOI: 10.4103/jrpp.jrpp_20_65

Castellani R, Ristic C, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a multicentre analysis". Contemp Clin Trials 32(2017): 432-443. DOI: 10.3389/fdgth.2026.1763884

Dumitrescu A, Andric D, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a prospective cohort study". Ther Innov Regul Sci 34(2020): 443-455. DOI: 10.2196/48297

Eriksdottir B, Brankovic E, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a randomised evaluation". Br J Clin Pharmacol 36(2023): 454-467. DOI: 10.1007/s00330-025-12031-z

Farkas I, Ionescu F, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a consensus statement". PLoS Med 38(2011): 465-479. DOI: 10.3389/fdgth.2026.1699005

Grabowski P, Petrescu G, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a retrospective analysis". Lancet 40(2014): 476-482. DOI: 10.3389/fdgth.2026.1834800

Hendriksen W, Wojcik H, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a validation study". N Engl J Med 42(2017): 487-494. DOI: 10.2196/69678

Ionescu F, Foscarini I, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a scoping review". JAMA 44(2020): 498-506. DOI: 10.7759/cureus.46454

Jankowiak G, Grabowski J, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a health-economic analysis". Nat Med 46(2023): 509-518. DOI: 10.1016/j.ssci.2025.107028

Kowalczyk N, Novotna K, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a systematic review". Cell Rep Med 8(2011): 520-530. DOI: 10.2196/48633

Lindqvist U, Urbanek L, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a methodological appraisal". Clin Transl Sci 10(2014): 531-542. DOI: 10.1371/journal.pone.0305949

Marchetti D, Doncheva M, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a multicentre analysis". Pharmacogenomics J 12(2017): 542-554. DOI: 10.1371/journal.pdig.0000514

Novotna E, Eriksdottir N, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a prospective cohort study". Regul Toxicol Pharmacol 14(2020): 553-566. DOI: 10.17219/acem/208596

Ostrowski L, Lindqvist O, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a randomised evaluation". Drug Saf 16(2023): 564-578. DOI: 10.7759/cureus.104086

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Ristic C, Castellani R, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a validation study". Sci Transl Med 22(2017): 597-605. DOI: 10.1177/08404704241291226

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Wojcik H, Hendriksen W, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a multicentre analysis". Drug Discov Today 32(2017): 652-665. DOI: 10.3390/diagnostics15162117

Zielinski O, Ostrowski Z, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a prospective cohort study". Clin Cancer Res 34(2020): 663-677. DOI: 10.4103/jrpp.jrpp_20_65

Andric V, Vasquez A, et al. "CRISPR Base Editing for a Monogenic Liver Disorder: a randomised evaluation". BMJ Open 36(2023): 674-680. DOI: 10.3389/fdgth.2026.1763884

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Published

2024-08-13

How to Cite

CRISPR Base Editing for a Monogenic Liver Disorder: Off-Target Assessment and Long-Term Follow-Up Obligations. (2024). Translational Medicine & Therapeutics Journal, 3(2), 74-82. https://doi.org/10.5281/zenodo.22649285

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