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Items: 1 to 20 of 138

1.

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.

2.

Gene therapy strategies for X-linked adrenoleukodystrophy.

Cartier N.

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

PMID:
11525559
3.

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
4.

Therapy of X-linked adrenoleukodystrophy.

Semmler A, Köhler W, Jung HH, Weller M, Linnebank M.

Expert Rev Neurother. 2008 Sep;8(9):1367-79. doi: 10.1586/14737175.8.9.1367. Review.

5.

X-linked adrenoleukodystrophy.

Moser HW, Mahmood A, Raymond GV.

Nat Clin Pract Neurol. 2007 Mar;3(3):140-51. Review.

PMID:
17342190
6.
7.

X-linked adrenoleukodystrophy: therapeutic approaches to distinct phenotypes.

Mahmood A, Dubey P, Moser HW, Moser A.

Pediatr Transplant. 2005 Dec;9 Suppl 7:55-62. Review.

PMID:
16305618
8.

[Hematopoietic cell transplantation in X-linked adrenoleukodystrophy].

Kato S.

Brain Nerve. 2007 Apr;59(4):339-46. Review. Japanese.

PMID:
17447520
9.

ABCD1 mutations and the X-linked adrenoleukodystrophy mutation database: role in diagnosis and clinical correlations.

Kemp S, Pujol A, Waterham HR, van Geel BM, Boehm CD, Raymond GV, Cutting GR, Wanders RJ, Moser HW.

Hum Mutat. 2001 Dec;18(6):499-515. Review.

PMID:
11748843
10.

Stem cell directed gene therapy.

Engel BC, Kohn DB.

Front Biosci. 1999 May 1;4:e26-33. Review.

PMID:
10228093
11.

X-linked adrenoleukodystrophy: very long-chain fatty acid metabolism, ABC half-transporters and the complicated route to treatment.

Kemp S, Wanders RJ.

Mol Genet Metab. 2007 Mar;90(3):268-76. Epub 2006 Nov 7. Review.

PMID:
17092750
12.

Cerebral inflammation in X-linked adrenoleukodystrophy.

McGuinness MC, Smith KD.

Arch Immunol Ther Exp (Warsz). 1999;47(5):281-7. Review.

PMID:
10604233
13.

X-linked adrenoleukodystrophy: diagnostic and follow-up system in Japan.

Shimozawa N, Honda A, Kajiwara N, Kozawa S, Nagase T, Takemoto Y, Suzuki Y.

J Hum Genet. 2011 Feb;56(2):106-9. doi: 10.1038/jhg.2010.139. Epub 2010 Nov 11. Review.

PMID:
21068741
14.

Hematopoietic stem cells in HIV disease.

Scadden DT, Shen H, Cheng T.

J Natl Cancer Inst Monogr. 2001;(28):24-9. Review.

PMID:
11158203
15.

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
16.

Human immunodeficiency virus and the hematopoietic repertoire: implications for gene therapy.

Panoskaltsis N, Abboud CN.

Front Biosci. 1999 May 1;4:D457-67. Review.

PMID:
10228094
17.

Stem cell engineering for the treatment of severe hemoglobinopathies.

Sadelain M, Boulad F, Lisowki L, Moi P, Riviere I.

Curr Mol Med. 2008 Nov;8(7):690-7. Review.

PMID:
18991654
18.

Gene transfer to hematopoietic cells--the clinical experience.

Richter J.

Eur J Haematol. 1997 Aug;59(2):67-75. Review. No abstract available.

PMID:
9293853
19.

Pathophysiology of X-linked adrenoleukodystrophy.

Berger J, Forss-Petter S, Eichler FS.

Biochimie. 2014 Mar;98:135-42. doi: 10.1016/j.biochi.2013.11.023. Epub 2013 Dec 4. Review.

20.

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