Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates. [artículo]
Por: Enríquez de Salamanca Lorente, Rafael [Instituto de Investigación i+12]
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Colaborador(es): Instituto de Investigación imas12.
Editor: Journal of Translational Medicine, 2012Descripción: 10:122.Recursos en línea: Acceso libre Resumen: Adeno-associated vectors (rAAV) have been used to attain long-term liver gene expression. In humans, the cellular immune response poses a serious obstacle for transgene persistence while neutralizing humoral immunity curtails re-administration. Porphobilinogen deaminase (PBGD) haploinsufficiency (acute intermittent porphyria) benefits from liver gene transfer in mouse models and clinical trials are about to begin. In this work, we sought to study in non-human primates the feasibility of repeated gene-transfer with intravenous administration of rAAV5 vectors under the effects of an intensive immunosuppressive regimen and to analyze its ability to circumvent T-cell immunity and thereby prolong transgene expression.
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Artículo
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Formato Vancouver:
Unzu C, Hervás-Stubbs S, Sampedro A, Mauleón I, Mancheño U, Alfaro C, et al. Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates. J Transl Med. 2012 Jun
15;10:122.
PMID: 22704060
Contiene 26 referencias
Adeno-associated vectors (rAAV) have been used to attain long-term liver gene expression. In humans, the cellular immune response poses a serious obstacle for transgene persistence while neutralizing humoral immunity curtails re-administration. Porphobilinogen deaminase (PBGD) haploinsufficiency (acute intermittent porphyria) benefits from liver gene transfer in mouse models and clinical trials are about to begin. In this work, we sought to study in non-human primates the feasibility of repeated gene-transfer with intravenous administration of rAAV5 vectors under the effects of an intensive immunosuppressive regimen and to analyze its ability to circumvent T-cell immunity and thereby prolong transgene expression.
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