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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: mitrophanous ka. 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.
Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery.
Mazarakis ND, Azzouz M, Rohll JB, Ellard FM, Wilkes FJ, Olsen AL, Carter EE, Barber RD, Baban DF, Kingsman SM, Kingsman AJ, O'Malley K, Mitrophanous KA. Mazarakis ND, et al. Among authors: mitrophanous ka. Hum Mol Genet. 2001 Sep 15;10(19):2109-21. doi: 10.1093/hmg/10.19.2109. Hum Mol Genet. 2001. PMID: 11590128
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: mitrophanous ka. 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: mitrophanous ka. 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.
62 results