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Items: 1 to 20 of 35


Unusual brain images of a boy with adolescent cerebral X-linked adrenoleukodystrophy presenting with exhibitionism: A CARE-compliant case report.

Zheng F, Lin Z, Ye X, Shi X.

Medicine (Baltimore). 2017 Dec;96(51):e9481. doi: 10.1097/MD.0000000000009481.


Childhood cerebral X-linked adrenoleukodystrophy with atypical neuroimaging abnormalities and a novel mutation.

Muranjan M, Karande S, Sankhe S, Eichler S.

J Postgrad Med. 2018 Jan-Mar;64(1):59-63. doi: 10.4103/jpgm.JPGM_349_17.


Application of a diagnostic methodology by quantification of 26:0 lysophosphatidylcholine in dried blood spots for Japanese newborn screening of X-linked adrenoleukodystrophy.

Wu C, Iwamoto T, Igarashi J, Miyajima T, Hossain MA, Yanagisawa H, Akiyama K, Shintaku H, Eto Y.

Mol Genet Metab Rep. 2017 Jul 11;12:115-118. doi: 10.1016/j.ymgmr.2017.06.004. eCollection 2017 Sep.


Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point of View.

Cavazzana M, Ribeil JA, Lagresle-Peyrou C, André-Schmutz I.

Stem Cells Dev. 2017 Jan 15;26(2):71-76. doi: 10.1089/scd.2016.0230. Epub 2016 Oct 16. Review.


Adenoassociated virus serotype 9-mediated gene therapy for x-linked adrenoleukodystrophy.

Gong Y, Mu D, Prabhakar S, Moser A, Musolino P, Ren J, Breakefield XO, Maguire CA, Eichler FS.

Mol Ther. 2015 May;23(5):824-834. doi: 10.1038/mt.2015.6. Epub 2015 Jan 16.


ABCD1 deletion-induced mitochondrial dysfunction is corrected by SAHA: implication for adrenoleukodystrophy.

Baarine M, Beeson C, Singh A, Singh I.

J Neurochem. 2015 May;133(3):380-96. doi: 10.1111/jnc.12992. Epub 2015 Jan 13.


Recent trends of polymer mediated liposomal gene delivery system.

Kundu SK, Sharma AR, Lee SS, Sharma G, Doss CG, Yagihara S, Kim DY, Nam JS, Chakraborty C.

Biomed Res Int. 2014;2014:934605. doi: 10.1155/2014/934605. Epub 2014 Aug 27. Review.


Evaluation of retinoids for induction of the redundant gene ABCD2 as an alternative treatment option in X-linked adrenoleukodystrophy.

Weber FD, Weinhofer I, Einwich A, Forss-Petter S, Muneer Z, Maier H, Weber WH, Berger J.

PLoS One. 2014 Jul 31;9(7):e103742. doi: 10.1371/journal.pone.0103742. eCollection 2014.


Transgene expression in various organs post BM-HSC transplantation.

Wang N, Rajasekaran N, Hou T, Mellins ED.

Stem Cell Res. 2014 Jan;12(1):209-21. doi: 10.1016/j.scr.2013.10.010. Epub 2013 Nov 2.


Lentiviral vectors for cancer immunotherapy and clinical applications.

Liechtenstein T, Perez-Janices N, Escors D.

Cancers (Basel). 2013 Sep;5(3):815-37. doi: 10.3390/cancers5030815.


Recent gene therapy advancements for neurological diseases.

Nagabhushan Kalburgi S, Khan NN, Gray SJ.

Discov Med. 2013 Feb;15(81):111-9. Review.


ABCD2 is a direct target of β-catenin and TCF-4: implications for X-linked adrenoleukodystrophy therapy.

Park CY, Kim HS, Jang J, Lee H, Lee JS, Yoo JE, Lee DR, Kim DW.

PLoS One. 2013;8(2):e56242. doi: 10.1371/journal.pone.0056242. Epub 2013 Feb 21.


Oxidative stress modulates mitochondrial failure and cyclophilin D function in X-linked adrenoleukodystrophy.

López-Erauskin J, Galino J, Bianchi P, Fourcade S, Andreu AL, Ferrer I, Muñoz-Pinedo C, Pujol A.

Brain. 2012 Dec;135(Pt 12):3584-98. doi: 10.1093/brain/aws292.


Intravenous immunoglobulin treatment in a patient with adrenomyeloneuropathy.

Jønch AE, Danielsen ER, Thomsen C, Meden P, Svenstrup K, Nielsen JE.

BMC Neurol. 2012 Sep 26;12:108. doi: 10.1186/1471-2377-12-108.


Adrenoleukodystrophy: a forgotten diagnosis in children with primary Addison's disease.

Nascimento M, Rodrigues N, Espada F, Fonseca M.

BMJ Case Rep. 2012 Aug 21;2012. pii: bcr2012006308. doi: 10.1136/bcr-2012-006308.


Bezafibrate for X-linked adrenoleukodystrophy.

Engelen M, Tran L, Ofman R, Brennecke J, Moser AB, Dijkstra IM, Wanders RJ, Poll-The BT, Kemp S.

PLoS One. 2012;7(7):e41013. doi: 10.1371/journal.pone.0041013. Epub 2012 Jul 20.


X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management.

Engelen M, Kemp S, de Visser M, van Geel BM, Wanders RJ, Aubourg P, Poll-The BT.

Orphanet J Rare Dis. 2012 Aug 13;7:51. doi: 10.1186/1750-1172-7-51.


Activation of the stress proteome as a mechanism for small molecule therapeutics.

Brose RD, Shin G, McGuinness MC, Schneidereith T, Purvis S, Dong GX, Keefer J, Spencer F, Smith KD.

Hum Mol Genet. 2012 Oct 1;21(19):4237-52. doi: 10.1093/hmg/dds247. Epub 2012 Jul 2.

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