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AAV9-mediated engineering of autotransplanted kidney of non-human primates

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AAV9-mediated engineering of autotransplanted kidney of non-human primates. / Tomasoni, S.; Trionfini, P.; Azzollini, N.; Zentilin, L.; Giacca, M.; Aiello, S.; Longaretti, L.; Cozzi, E.; Baldan, N.; Remuzzi, G.; Benigni, A.

In: Gene Therapy, Vol. 24, No. 5, 01.05.2017, p. 308-313.

Research output: Contribution to journalArticle

Harvard

Tomasoni, S, Trionfini, P, Azzollini, N, Zentilin, L, Giacca, M, Aiello, S, Longaretti, L, Cozzi, E, Baldan, N, Remuzzi, G & Benigni, A 2017, 'AAV9-mediated engineering of autotransplanted kidney of non-human primates', Gene Therapy, vol. 24, no. 5, pp. 308-313. https://doi.org/10.1038/gt.2017.21

APA

Tomasoni, S., Trionfini, P., Azzollini, N., Zentilin, L., Giacca, M., Aiello, S., Longaretti, L., Cozzi, E., Baldan, N., Remuzzi, G., & Benigni, A. (2017). AAV9-mediated engineering of autotransplanted kidney of non-human primates. Gene Therapy, 24(5), 308-313. https://doi.org/10.1038/gt.2017.21

Vancouver

Tomasoni S, Trionfini P, Azzollini N, Zentilin L, Giacca M, Aiello S et al. AAV9-mediated engineering of autotransplanted kidney of non-human primates. Gene Therapy. 2017 May 1;24(5):308-313. https://doi.org/10.1038/gt.2017.21

Author

Tomasoni, S. ; Trionfini, P. ; Azzollini, N. ; Zentilin, L. ; Giacca, M. ; Aiello, S. ; Longaretti, L. ; Cozzi, E. ; Baldan, N. ; Remuzzi, G. ; Benigni, A. / AAV9-mediated engineering of autotransplanted kidney of non-human primates. In: Gene Therapy. 2017 ; Vol. 24, No. 5. pp. 308-313.

Bibtex Download

@article{55da512d2be5454a95c9c7c33ec154e1,
title = "AAV9-mediated engineering of autotransplanted kidney of non-human primates",
abstract = "Ex vivo gene transfer to the graft before transplantation is an attractive option for circumventing systemic side effects of chronic antirejection therapy. Gene delivery of the immunomodulatory protein cytotoxic T-lymphocyte-associated protein 4-immunoglobulin (CTLA4-Ig) prevented chronic kidney rejection in a rat model of allotransplantation without the need for systemic immunosuppression. Here we generated adeno-associated virus type 2 (AAV2) and AAV9 vectors encoding for LEA29Y, an optimized version of CTLA4-Ig. Both LEA29Y vectors were equally efficient for reducing T-cell proliferation in vitro. Serotype 9 was chosen for in vivo experiments owing to a lower frequency of preformed antibodies against the AAV9 capsid in 16 non-human primate tested sera. AAV9-LEA29Y was able to transduce the kidney of non-human primates in an autotransplantation model. Expression of LEA29Y mRNA by renal cells translated into the production of the corresponding protein, which was confined to the graft but not detected in serum. Results in non-human primates represent a step forward in maintaining the portability of this strategy into clinics.",
author = "S. Tomasoni and P. Trionfini and N. Azzollini and L. Zentilin and M. Giacca and S. Aiello and L. Longaretti and E. Cozzi and N. Baldan and G. Remuzzi and A. Benigni",
year = "2017",
month = may,
day = "1",
doi = "10.1038/gt.2017.21",
language = "English",
volume = "24",
pages = "308--313",
journal = "Gene Therapy",
issn = "0969-7128",
publisher = "Nature Publishing Group",
number = "5",

}

RIS (suitable for import to EndNote) Download

TY - JOUR

T1 - AAV9-mediated engineering of autotransplanted kidney of non-human primates

AU - Tomasoni, S.

AU - Trionfini, P.

AU - Azzollini, N.

AU - Zentilin, L.

AU - Giacca, M.

AU - Aiello, S.

AU - Longaretti, L.

AU - Cozzi, E.

AU - Baldan, N.

AU - Remuzzi, G.

AU - Benigni, A.

PY - 2017/5/1

Y1 - 2017/5/1

N2 - Ex vivo gene transfer to the graft before transplantation is an attractive option for circumventing systemic side effects of chronic antirejection therapy. Gene delivery of the immunomodulatory protein cytotoxic T-lymphocyte-associated protein 4-immunoglobulin (CTLA4-Ig) prevented chronic kidney rejection in a rat model of allotransplantation without the need for systemic immunosuppression. Here we generated adeno-associated virus type 2 (AAV2) and AAV9 vectors encoding for LEA29Y, an optimized version of CTLA4-Ig. Both LEA29Y vectors were equally efficient for reducing T-cell proliferation in vitro. Serotype 9 was chosen for in vivo experiments owing to a lower frequency of preformed antibodies against the AAV9 capsid in 16 non-human primate tested sera. AAV9-LEA29Y was able to transduce the kidney of non-human primates in an autotransplantation model. Expression of LEA29Y mRNA by renal cells translated into the production of the corresponding protein, which was confined to the graft but not detected in serum. Results in non-human primates represent a step forward in maintaining the portability of this strategy into clinics.

AB - Ex vivo gene transfer to the graft before transplantation is an attractive option for circumventing systemic side effects of chronic antirejection therapy. Gene delivery of the immunomodulatory protein cytotoxic T-lymphocyte-associated protein 4-immunoglobulin (CTLA4-Ig) prevented chronic kidney rejection in a rat model of allotransplantation without the need for systemic immunosuppression. Here we generated adeno-associated virus type 2 (AAV2) and AAV9 vectors encoding for LEA29Y, an optimized version of CTLA4-Ig. Both LEA29Y vectors were equally efficient for reducing T-cell proliferation in vitro. Serotype 9 was chosen for in vivo experiments owing to a lower frequency of preformed antibodies against the AAV9 capsid in 16 non-human primate tested sera. AAV9-LEA29Y was able to transduce the kidney of non-human primates in an autotransplantation model. Expression of LEA29Y mRNA by renal cells translated into the production of the corresponding protein, which was confined to the graft but not detected in serum. Results in non-human primates represent a step forward in maintaining the portability of this strategy into clinics.

UR - http://www.scopus.com/inward/record.url?scp=85017433130&partnerID=8YFLogxK

U2 - 10.1038/gt.2017.21

DO - 10.1038/gt.2017.21

M3 - Article

C2 - 28346435

AN - SCOPUS:85017433130

VL - 24

SP - 308

EP - 313

JO - Gene Therapy

JF - Gene Therapy

SN - 0969-7128

IS - 5

ER -

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