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Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.
Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, Vidaud M, Abel U, Dal-Cortivo L, Caccavelli L, Mahlaoui N, Kiermer V, Mittelstaedt D, Bellesme C, Lahlou N, Lefrère F, Blanche S, Audit M, Payen E, Leboulch P, l'Homme B, Bougnères P, Von Kalle C, Fischer A, Cavazzana-Calvo M, Aubourg P. Cartier N, et al. Among authors: aubourg p. Science. 2009 Nov 6;326(5954):818-23. doi: 10.1126/science.1171242. Science. 2009. PMID: 19892975
Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy.
Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Bougnères P, Schmidt M, Kalle CV, Fischer A, Cavazzana-Calvo M, Aubourg P. Cartier N, et al. Among authors: aubourg p. Methods Enzymol. 2012;507:187-98. doi: 10.1016/B978-0-12-386509-0.00010-7. Methods Enzymol. 2012. PMID: 22365775 Clinical Trial.
Gene therapy for leukodystrophies.
Biffi A, Aubourg P, Cartier N. Biffi A, et al. Among authors: aubourg p. Hum Mol Genet. 2011 Apr 15;20(R1):R42-53. doi: 10.1093/hmg/ddr142. Epub 2011 Mar 31. Hum Mol Genet. 2011. PMID: 21459776 Review.
182 results