000			01780na a2200229 4500
003			H12O
005			20210625062803.0
008			130622s2012 xxx||||| |||| 00| 0 eng d
040			_cH12O
041			_aeng
100			_9763 _aEnríquez de Salamanca Lorente, Rafael _eInstituto de Investigación i+12
245	0	0	_aTransient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates. _h[artículo]
260			_bJournal of Translational Medicine, _c2012
300			_a10:122.
500			_aFormato 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.
501			_aPMID: 22704060
504			_aContiene 26 referencias
520			_aAdeno-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.
710			_9625 _aInstituto de Investigación imas12
856			_uhttp://www.ncbi.nlm.nih.gov/pmc/articles/PMC3412719/ _yAcceso libre
942			_n0 _2ddc _cART
999			_c6594 _d6594