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Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.
Palfi S, Gurruchaga JM, Ralph GS, Lepetit H, Lavisse S, Buttery PC, Watts C, Miskin J, Kelleher M, Deeley S, Iwamuro H, Lefaucheur JP, Thiriez C, Fenelon G, Lucas C, Brugières P, Gabriel I, Abhay K, Drouot X, Tani N, Kas A, Ghaleh B, Le Corvoisier P, Dolphin P, Breen DP, Mason S, Guzman NV, Mazarakis ND, Radcliffe PA, Harrop R, Kingsman SM, Rascol O, Naylor S, Barker RA, Hantraye P, Remy P, Cesaro P, Mitrophanous KA. Palfi S, et al. Among authors: mazarakis nd. Lancet. 2014 Mar 29;383(9923):1138-46. doi: 10.1016/S0140-6736(13)61939-X. Epub 2014 Jan 10. Lancet. 2014. PMID: 24412048 Clinical Trial.
Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia.
Jarraya B, Boulet S, Ralph GS, Jan C, Bonvento G, Azzouz M, Miskin JE, Shin M, Delzescaux T, Drouot X, Hérard AS, Day DM, Brouillet E, Kingsman SM, Hantraye P, Mitrophanous KA, Mazarakis ND, Palfi S. Jarraya B, et al. Among authors: mazarakis nd. Sci Transl Med. 2009 Oct 14;1(2):2ra4. doi: 10.1126/scitranslmed.3000130. Sci Transl Med. 2009. PMID: 20368163
New methods to titrate EIAV-based lentiviral vectors.
Martin-Rendon E, White LJ, Olsen A, Mitrophanous KA, Mazarakis ND. Martin-Rendon E, et al. Among authors: mazarakis nd. Mol Ther. 2002 May;5(5 Pt 1):566-70. doi: 10.1006/mthe.2002.0576. Mol Ther. 2002. PMID: 11991747 Free article.
Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease.
Azzouz M, Martin-Rendon E, Barber RD, Mitrophanous KA, Carter EE, Rohll JB, Kingsman SM, Kingsman AJ, Mazarakis ND. Azzouz M, et al. Among authors: mazarakis nd. J Neurosci. 2002 Dec 1;22(23):10302-12. doi: 10.1523/JNEUROSCI.22-23-10302.2002. J Neurosci. 2002. PMID: 12451130 Free PMC article.
64 results