Systemic delivery of high doses of adeno-associated viral (AAV) vectors causes insertional mutagenesis and hepatocellular carcinomas in newborn mice. The study shows that this risk is lower in young adult mice and juvenile cats using AAV doses similar to those used in many clinical applications.

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors / Ferla, R.; Alliegro, M.; Dell'Anno, M.; Nusco, E.; Cullen, J. M.; Smith, S. N.; Wolfsberg, T. G.; O'Donnell, P.; Wang, P.; Nguyen, A. -D.; Chandler, R. J.; Chen, Z.; Burgess, S. M.; Vite, C. H.; Haskins, M. E.; Venditti, C. P.; Auricchio, A.. - In: MOLECULAR THERAPY. METHODS & CLINICAL DEVELOPMENT. - ISSN 2329-0501. - 20:(2021), pp. 247-257. [10.1016/j.omtm.2020.11.015]

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors

Auricchio A.
2021

Abstract

Systemic delivery of high doses of adeno-associated viral (AAV) vectors causes insertional mutagenesis and hepatocellular carcinomas in newborn mice. The study shows that this risk is lower in young adult mice and juvenile cats using AAV doses similar to those used in many clinical applications.
2021
Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors / Ferla, R.; Alliegro, M.; Dell'Anno, M.; Nusco, E.; Cullen, J. M.; Smith, S. N.; Wolfsberg, T. G.; O'Donnell, P.; Wang, P.; Nguyen, A. -D.; Chandler, R. J.; Chen, Z.; Burgess, S. M.; Vite, C. H.; Haskins, M. E.; Venditti, C. P.; Auricchio, A.. - In: MOLECULAR THERAPY. METHODS & CLINICAL DEVELOPMENT. - ISSN 2329-0501. - 20:(2021), pp. 247-257. [10.1016/j.omtm.2020.11.015]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11588/854859
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