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Results: 1 to 20 of 97

1.

Gene therapy starts to tackle CNS disease.

Aubourg P.

Rinsho Shinkeigaku. 2010 Nov;50(11):842. No abstract available.

PMID:
21921466
[PubMed - indexed for MEDLINE]
2.

Medicine. A comeback for gene therapy.

Naldini L.

Science. 2009 Nov 6;326(5954):805-6. doi: 10.1126/science.1181937. No abstract available.

PMID:
19892968
[PubMed - indexed for MEDLINE]
3.

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.

Science. 2009 Nov 6;326(5954):818-23. doi: 10.1126/science.1171242.

PMID:
19892975
[PubMed - indexed for MEDLINE]
Free Article
4.

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.

Methods Enzymol. 2012;507:187-98. doi: 10.1016/B978-0-12-386509-0.00010-7.

PMID:
22365775
[PubMed - indexed for MEDLINE]
5.

Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice.

Benhamida S, Pflumio F, Dubart-Kupperschmitt A, Zhao-Emonet JC, Cavazzana-Calvo M, Rocchiccioli F, Fichelson S, Aubourg P, Charneau P, Cartier N.

Mol Ther. 2003 Mar;7(3):317-24.

PMID:
12668127
[PubMed - indexed for MEDLINE]
6.

[Gene therapy of x-linked adrenoleukodystrophy using hematopoietic stem cells and a lentiviral vector].

Cartier N, Hacein-Bey-Abina S, Von Kalle C, Bougnères P, Fischer A, Cavazzana-Calvo M, Aubourg P.

Bull Acad Natl Med. 2010 Feb;194(2):255-64; discussion 264-8. French.

PMID:
21166117
[PubMed - indexed for MEDLINE]
7.

Adrenoleukodystrophy patient perspective: turning despair into a gene therapy breakthrough.

Salzman A.

Hum Gene Ther. 2011 Jun;22(6):647-8. doi: 10.1089/hum.2011.2503. No abstract available.

PMID:
21651376
[PubMed - indexed for MEDLINE]
8.

Gene therapy for leukodystrophies.

Biffi A, Aubourg P, Cartier N.

Hum Mol Genet. 2011 Apr 15;20(R1):R42-53. doi: 10.1093/hmg/ddr142. Epub 2011 Mar 31. Review.

PMID:
21459776
[PubMed - indexed for MEDLINE]
Free Article
9.

Hematopoietic stem cell gene therapy in Hurler syndrome, globoid cell leukodystrophy, metachromatic leukodystrophy and X-adrenoleukodystrophy.

Cartier N, Aubourg P.

Curr Opin Mol Ther. 2008 Oct;10(5):471-8. Review.

PMID:
18830923
[PubMed - indexed for MEDLINE]
10.

[Gene therapy in the adrenoleukodystrophy].

Nau JY.

Rev Med Suisse. 2009 Nov 18;5(226):2363. French. No abstract available.

PMID:
20052872
[PubMed - indexed for MEDLINE]
11.

Gene therapy strategies for X-linked adrenoleukodystrophy.

Cartier N.

Curr Opin Mol Ther. 2001 Aug;3(4):357-61. Review.

PMID:
11525559
[PubMed - indexed for MEDLINE]
12.

Co-expression of mutated and normal adrenoleukodystrophy protein reduces protein function: implications for gene therapy of X-linked adrenoleukodystrophy.

Unterrainer G, Molzer B, Forss-Petter S, Berger J.

Hum Mol Genet. 2000 Nov 1;9(18):2609-16.

PMID:
11063720
[PubMed - indexed for MEDLINE]
Free Article
13.

X-linked adrenoleukodystrophy.

Aubourg P, Mandel JL.

Ann N Y Acad Sci. 1996 Dec 27;804:461-76. Review. No abstract available.

PMID:
8993565
[PubMed - indexed for MEDLINE]
14.

Lentiviruses as vectors for CNS diseases.

Déglon N, Aebischer P.

Curr Top Microbiol Immunol. 2002;261:191-209. Review. No abstract available.

PMID:
11892248
[PubMed - indexed for MEDLINE]
15.

Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders.

Persons DA.

Hematology Am Soc Hematol Educ Program. 2009:690-7. doi: 10.1182/asheducation-2009.1.690. Review.

PMID:
20008255
[PubMed - indexed for MEDLINE]
Free Article
16.

[Ten years of gene therapy: thoughts and perspectives].

Cavazzana-Calvo M, Hacein-Bey-Abina S, Fischer A.

Med Sci (Paris). 2010 Feb;26(2):115-8. doi: 10.1051/medsci/2010262115. French. No abstract available.

PMID:
20188032
[PubMed - indexed for MEDLINE]
Free Article
17.

X-linked adrenoleukodystrophy enigma: how does the ALD peroxisomal transporter mutation affect CNS glia?

Aubourg P, Dubois-Dalcq M.

Glia. 2000 Jan 15;29(2):186-90. Review. No abstract available.

PMID:
10625338
[PubMed - indexed for MEDLINE]
18.

Opportunities for the use of lentiviral vectors in human gene therapy.

Galimi F, Verma IM.

Curr Top Microbiol Immunol. 2002;261:245-54. Review. No abstract available.

PMID:
11892251
[PubMed - indexed for MEDLINE]
19.

Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy.

Cartier N, Aubourg P.

Brain Pathol. 2010 Jul;20(4):857-62. doi: 10.1111/j.1750-3639.2010.00394.x. Review.

PMID:
20626747
[PubMed - indexed for MEDLINE]
20.

Targeted inactivation of the X-linked adrenoleukodystrophy gene in mice.

Forss-Petter S, Werner H, Berger J, Lassmann H, Molzer B, Schwab MH, Bernheimer H, Zimmermann F, Nave KA.

J Neurosci Res. 1997 Dec 1;50(5):829-43.

PMID:
9418970
[PubMed - indexed for MEDLINE]

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