Gene Therapy in a Patient With Pre-Existing Vector Immunity: A Two-Case Series of Alternative Serotypes

Authors

  • Susanne Stellbrink Department of Cell and Gene Therapy, Hunsruck Institute of Translational Medicine, Idar-Oberstein, Germany Author
  • Bence Tarjanyi Centre for Translational Immunology, Orseg Centre for Experimental Therapeutics, Szentgotthard, Hungary Author
  • Ignacio Villaescusa Department of Rare Disease Medicine, Alcarria Centre for Experimental Therapeutics, Guadalajara, Spain Author

DOI:

https://doi.org/10.5281/zenodo.22649434

Keywords:

Gene Therapy in a Patient With Pre-Existing Vector Immunity, Translational medicine; Evidence synthesis;, Clinical implementation; Health services research

Abstract

Gene Therapy in a Patient With Pre-Existing Vector Immunity 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 gene therapy in
a patient with pre-existing vector immunity, 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.

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Castellani R, Ristic C, et al. "Gene Therapy in a Patient With Pre-Existing Vector Immunity: a multicentre analysis". Contemp Clin Trials 32(2017): 432-443. DOI: 10.1371/journal.pdig.0000514

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Eriksdottir B, Brankovic E, et al. "Gene Therapy in a Patient With Pre-Existing Vector Immunity: a randomised evaluation". Br J Clin Pharmacol 36(2023): 454-467. DOI: 10.7759/cureus.104086

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Grabowski P, Petrescu G, et al. "Gene Therapy in a Patient With Pre-Existing Vector Immunity: a retrospective analysis". Lancet 40(2014): 476-482. DOI: 10.1016/j.mayocpiqo.2022.09.004

Hendriksen W, Wojcik H, et al. "Gene Therapy in a Patient With Pre-Existing Vector Immunity: a validation study". N Engl J Med 42(2017): 487-494. DOI: 10.1177/08404704241291226

Ionescu F, Foscarini I, et al. "Gene Therapy in a Patient With Pre-Existing Vector Immunity: a scoping review". JAMA 44(2020): 498-506. DOI: 10.1093/medlaw/fwad013

Jankowiak G, Grabowski J, et al. "Gene Therapy in a Patient With Pre-Existing Vector Immunity: a health-economic analysis". Nat Med 46(2023): 509-518. DOI: 10.2196/89834

Kowalczyk N, Novotna K, et al. "Gene Therapy in a Patient With Pre-Existing Vector Immunity: a systematic review". Cell Rep Med 8(2011): 520-530. DOI: 10.1007/s10916-024-02104-9

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Wojcik H, Hendriksen W, et al. "Gene Therapy in a Patient With Pre-Existing Vector Immunity: a multicentre analysis". Drug Discov Today 32(2017): 652-665. DOI: 10.2196/48633

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Andric V, Vasquez A, et al. "Gene Therapy in a Patient With Pre-Existing Vector Immunity: a randomised evaluation". BMJ Open 36(2023): 674-680. DOI: 10.1371/journal.pdig.0000514

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Published

2025-11-11

How to Cite

Gene Therapy in a Patient With Pre-Existing Vector Immunity: A Two-Case Series of Alternative Serotypes. (2025). Translational Medicine & Therapeutics Journal, 4(2), 92-100. https://doi.org/10.5281/zenodo.22649434

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