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 |