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. 2012 Sep 5;3(2):246–253. doi: 10.1016/j.nurx.2006.01.004

Therapy of X-linked adrenoleukodystrophy

Hugo W Moser 1,2,✉
PMCID: PMC3593437  PMID: 16554262

Summary

Current therapies for X-linked adrenoleukodystrophy (X-ALD) include replacement therapy with adrenal steroids, which is mandatory for all patients with impaired adrenal function but does not alter neurological progression significantly; dietary therapy with “Lorenzo’s Oil,” which appears to have a preventive effect in asymptomatic boys whose brain MRI is normal; and hematopoietic stem cell transplantation in patients in the early stage of the cerebral inflammatory phenotype. Application of these interventions requires careful assessment of the patients’ phenotype, which often changes over time. Family screening provides important opportunities for disease prevention.

Key Words: Adrenoleukodystrophy, adrenomyeloneuropathy, adrenal insufficiency, bone marrow transplant, very long chain fatty acids, peroxisomal disorders, neonatal screening

References

  • 1.Moser HW, Smith KD, Watkins PA, Powers J, Moser AB. X-linked adrenoleukodystrophy. In: Scriver CR, Beaudet AL, Sly WS, Valle D, editors. The Metabolic and Molecular Bases of Inherited Disease. New York: McGraw Hill; 2001. pp. 3257–3301. [Google Scholar]
  • 2.Mosser J, Douar AM, Saide CO, Kioschis P, Feil R, Moser H, Poustka AM, Mandel JL, Aubourg P. Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters. Nature. 1993;361:726–730. doi: 10.1038/361726a0. [DOI] [PubMed] [Google Scholar]
  • 3.Dean M, Hamon Y, Chimini G. The human ATP-binding cassette (ABC) transporter superfamily. J Lipid Res. 2001;42:1007–1017. [PubMed] [Google Scholar]
  • 4.Igarashi M, Schaumburg HH, Powers J, Kishmoto Y, Kolodny E, Suzuki K. Fatty acid abnormality in adrenoleukodystrophy. J Neurochem. 1976;26:851–860. doi: 10.1111/j.1471-4159.1976.tb04462.x. [DOI] [PubMed] [Google Scholar]
  • 5.Moser AB, Kreiter N, Bezman L, Lu S, Raymond GV, Naidu S, Moser HW. Plasma very long chain fatty acids in 3,000 peroxisome disease patients and 29,000 controls. Ann Neurol. 1999;45:100–110. doi: 10.1002/1531-8249(199901)45:1<100::AID-ART16>3.0.CO;2-U. [DOI] [PubMed] [Google Scholar]
  • 6.Lazo O, Contreras M, Hashmi M, Stanley W, Irazu C, Singh I. Peroxisomal lignoceroyl-CoA ligase deficiency in childhood adrenoleukodystrophy and adrenomyeloneuropathy. Proc Natl Acad Sci U S A. 1988;85:7647–7651. doi: 10.1073/pnas.85.20.7647. [DOI] [PMC free article] [PubMed] [Google Scholar]
  • 7.Singh I, Moser AE, Goldfischer S, Moser HW. Lignoceric acid is oxidized in the peroxisome: implications for the Zellweger cerebro-hepato-renal syndrome and adrenoleukodystrophy. Proc Natl Acad Sci U S A. 1984;81:4203–4207. doi: 10.1073/pnas.81.13.4203. [DOI] [PMC free article] [PubMed] [Google Scholar]
  • 8.McGuinness MC, Lu JF, Zhang HP, Dong GX, Heinzer AK, Watkins PA, Powers J, Smith KD. Role of ALDP (ABCD1) and mitochondria in X-linked adrenoleukodystrophy. Mol Cell Biol. 2003;23:744–53. doi: 10.1128/MCB.23.2.744-753.2003. [DOI] [PMC free article] [PubMed] [Google Scholar]
  • 9.Ho JK, Moser H, Kishimoto Y, Hamilton JA. Interactions of a very long chain fatty acid with model membranes and serum albumin. Implications for the pathogenesis of adrenoleukodystrophy. J Clin Invest. 1995;96:1455–1463. doi: 10.1172/JCI118182. [DOI] [PMC free article] [PubMed] [Google Scholar]
  • 10.Moser HW, Moser AB, Naidu S, Bergin A. Clinical aspects of adrenoleukodystrophy and adrenomyeloneuropathy. Dev Neurosci. 1991;13:254–261. doi: 10.1159/000112170. [DOI] [PubMed] [Google Scholar]
  • 11.Restuccia D, Di Lazzaro V, Valeriani M, Oliviero A, Le Pera D, Barba C, Cappa M, Bertini E, Di Capua M, Tonali P. Neurophysiologic follow-up of long-term dietary treatment in adult-onset adrenoleukodystrophy. Neurology. 1999;52:810–816. doi: 10.1212/WNL.52.4.810. [DOI] [PubMed] [Google Scholar]
  • 12.Bezman L, Moser AB, Raymond GV, Rinaldo P, Watkins PA, Smith KD, Kass NE, Moser HW. Adrenoleukodystrophy: incidence, new mutation rate, and results of extended family screening. Ann Neurol. 2001;49:512–517. doi: 10.1002/ana.101. [DOI] [PubMed] [Google Scholar]
  • 13.Kemp S, Pujol A, Waterham HR, van Geel BM, Boehm CD, Raymond GV, Cutting GR, Wanders RJ, Moser HW. ABCD1 mutations and the X-linked adrenoleukodystrophy mutation database: role in diagnosis and clinical correlations. Hum Mutat. 2001;18:499–515. doi: 10.1002/humu.1227. [DOI] [PubMed] [Google Scholar]
  • 14.Boehm CD, Cutting GR, Lachtermacher MB, Moser HW, Chong SS. Accurate DNA-based diagnostic and carrier testing for X-linked adrenoleukodystrophy. Mol Genet Metab. 1999;66:128–136. doi: 10.1006/mgme.1998.2779. [DOI] [PubMed] [Google Scholar]
  • 15.Kelley RI, Datta NS, Dobyns WB, Hajra AK, Moser AB, Noetzel MJ, Zackai EH, Moser HW. Neonatal adrenoleukodystrophy: new cases, biochemical studies, and differentiation from Zellweger and related peroxisomal polydystrophy syndromes. Am J Med Genet. 1986;23:869–901. doi: 10.1002/ajmg.1320230404. [DOI] [PubMed] [Google Scholar]
  • 16.Siemerling E, Creutzfeldt HG. Bronzekrankheit und Sklerosierende Encephalomyelitis. Arch Psychiatr Nervenkr. 1923;68:217–244. doi: 10.1007/BF01835678. [DOI] [Google Scholar]
  • 17.Schaumburg HH, Powers JM, Raine CS, Suzuki K, Richardson EP. Adrenoleukodystrophy. A clinical and pathological study of 17 cases. Arch Neurol. 1975;32:577–591. doi: 10.1001/archneur.1975.00490510033001. [DOI] [PubMed] [Google Scholar]
  • 18.Loes DJ, Fatemi A, Melhem ER, Gupte N, Bezman L, Moser HW, Raymond GV. Analysis of MRI patterns aids prediction of progression in X-linked adrenoleukodystrophy. Neurology. 2003;61:369–374. doi: 10.1212/01.WNL.0000079050.91337.83. [DOI] [PubMed] [Google Scholar]
  • 19.Schilder P. Zur Frage der Encephalitis Periaxialis Diffusa (Sogenannet Diffuse Sklerose) Neur Psych. 1913;15:359–376. doi: 10.1007/BF02895263. [DOI] [Google Scholar]
  • 20.Powers JM, Liu Y, Moser AB, Moser HW. The inflammatory myelinopathy of adreno-leukodystrophy: cells, effector molecules, and pathogenetic implications. J Neuropathol Exp Neurol. 1992;51:630–643. doi: 10.1097/00005072-199211000-00007. [DOI] [PubMed] [Google Scholar]
  • 21.Ito M, Blumberg BM, Mock DJ, Goodman AD, Moser AB, Moser HW, Smith KD, Powers JM. Potential environmental and host participants in the early white matter lesion of adreno-leukodystrophy: morphologic evidence for CD8 cytotoxic T cells, cytolysis of oligodendrocytes, and CD1-mediated lipid antigen presentation. J Neuropathol Exp Neurol. 2001;60:1004–1019. doi: 10.1093/jnen/60.10.1004. [DOI] [PubMed] [Google Scholar]
  • 22.Paintlia AS, Gilg AG, Khan M, Singh AK, Barbosa E, Singh I. Correlation of very long chain fatty acids accumulation and inflammatory disease progression in childhood ALD: Implications for potential therapies. Neurobiol Dis. 2003;14:425–439. doi: 10.1016/j.nbd.2003.08.013. [DOI] [PubMed] [Google Scholar]
  • 23.Kumar AJ, Rosenbaum AE, Naidu S, Wenger L, Citrin CM, Lindenberg R, Kim WS, Zinreich SJ, Molliver ME, Mayberg HS, Moser HW. Adrenoleukodystrophy: correlating MR imaging with CT. Radiology. 1987;165:497–504. doi: 10.1148/radiology.165.2.3659373. [DOI] [PubMed] [Google Scholar]
  • 24.Loes DJ, Hite S, Moser H, Stillman AE, Shapiro E, Lockman L, Latchaw RE, Krivit W. Adrenoleukodystrophy: a scoring method for brain MR observations. AJNR Am J Neuroradiol. 1994;15:1761–1766. [PMC free article] [PubMed] [Google Scholar]
  • 25.Eichler FS, Itoh R, Barker PB, Mori S, Garrett ES, van Zijl PC, Moser HW, Raymond GV, Melhem ER. Proton MR spectroscopic and diffusion tensor brain MR imaging in X-linked adrenoleukodystrophy: initial experience. Radiology. 2002;225:245–252. doi: 10.1148/radiol.2251011040. [DOI] [PubMed] [Google Scholar]
  • 26.Oz G, Tkac I, Charnas LR, Choi IY, Bjoraker KJ, Shapiro EG, Gruetter R. Assessment of adrenoleukodystrophy lesions by high field MRS in non-sedated pediatric patients. Neurology. 2005;64:434–441. doi: 10.1212/01.WNL.0000150906.52208.E7. [DOI] [PubMed] [Google Scholar]
  • 27.Cox C, Dubey P, Raymond GV, Mahmood A, Moser AB, Moser HW. Cognitive evaluation of neurologically asymptomatic boys with X-linked adrenoleukodystrophy.Arch Neurol in press, 2005. [DOI] [PubMed]
  • 28.Moser HW, Raymond GV, Lu SE, Muenz LR, Moser AB, Xu J, Jones RO, Loes DJ, Melhem ER, Dubey P, Bezman L, Brereton NH, Odone A. Follow-up of 89 Lorenzo’s Oil treated asymptomatic adrenoleukodystrophy patients. Arch Neurol. 2005;62:1073–1080. doi: 10.1001/archneur.62.7.1073. [DOI] [PubMed] [Google Scholar]
  • 29.Moser HW, Moser AB, Smith KD, Bergin A, Borel J, Shankroff J, Stine OC, Merette C, Ott J, Krivit W, Shapiro E. Adrenoleukodystrophy: phenotypic variability and implications for therapy. J Inherit Metab Dis. 1992;15:645–664. doi: 10.1007/BF01799621. [DOI] [PubMed] [Google Scholar]
  • 30.Moser HW, Loes DJ, Melhem ER, Raymond GV, Bezman L, Cox CS, Lu SE. X-Linked adrenoleukodystrophy: overview and prognosis as a function of age and brain magnetic resonance imaging abnormality. A study involving 372 patients. Neuropediatrics. 2000;31:227–239. doi: 10.1055/s-2000-9236. [DOI] [PubMed] [Google Scholar]
  • 31.Edwin D, Speedie LJ, Kohler W, Naidu S, Kruse B, Moser HW. Cognitive and brain magnetic resonance imaging findings in adrenomyeloneuropathy. Ann Neurol. 1996;40:675–678. doi: 10.1002/ana.410400419. [DOI] [PubMed] [Google Scholar]
  • 32.Griffin JW, Goren E, Schaumburg H, Engel WK, Loriaux L. Adrenomyeloneuropathy: a probable variant of adrenoleukodystrophy. I. Clinical and endocrinologic aspects. Neurology. 1977;27:1107–1113. doi: 10.1212/WNL.27.12.1107. [DOI] [PubMed] [Google Scholar]
  • 33.Budka H, Sluga E, Heiss WD. Spastic paraplegia associated with Addison’s disease: adult variant of adreno-leukodystrophy. J Neurol. 1976;213:237–250. doi: 10.1007/BF00312873. [DOI] [PubMed] [Google Scholar]
  • 34.Powers JM. Adreno-leukodystrophy (adreno-testiculo-leukomyelo-neuropathic-complex) Clin Neuropathol. 1985;4:181–199. [PubMed] [Google Scholar]
  • 35.Powers JM, DeCiero DP, Ito M, Moser AB, Moser HW. Adrenomyeloneuropathy: a neuropathologic review featuring its noninflammatory myelopathy. J Neuropathol Exp Neurol. 2000;59:89–102. doi: 10.1093/jnen/59.2.89. [DOI] [PubMed] [Google Scholar]
  • 36.Powers JM, DeCiero DP, Cox C, Richfield EK, Ito M, Moser AB, Moser HW. The dorsal root ganglia in adrenomyeloneuropathy: neuronal atrophy and abnormal mitochondria. J Neuropathol Exp Neurol. 2001;60:493–501. doi: 10.1093/jnen/60.5.493. [DOI] [PubMed] [Google Scholar]
  • 37.van Geel BM, Koelman JH, Barth PG, Ongerboer de Visser BW. Peripheral nerve abnormalities in adrenomyeloneuropathy: a clinical and electrodiagnostic study. Neurology. 1996;46:112–118. doi: 10.1212/WNL.46.1.112. [DOI] [PubMed] [Google Scholar]
  • 38.Kumar AJ, Kohler W, Kruse B, Naidu S, Bergin A, Edwin D, Moser HW. MR findings in adult-onset adrenoleukodystrophy. AJNR Am J Neuroradiol. 1995;16:1227–1237. [PMC free article] [PubMed] [Google Scholar]
  • 39.Dubey P, Fatemi A, Barker PB, Degaonkar M, Troeger M, Zackowski K, Bastian A, Smith SA, Pomper MG, Moser HW, Raymond GV. Spectroscopic evidence of cerebral axonopathy in patients with “pure” adrenomyeloneuropathy. Neurology. 2005;64:304–310. doi: 10.1212/01.WNL.0000149514.13580.84. [DOI] [PubMed] [Google Scholar]
  • 40.Fatemi A, Smith SA, Dubey P, Zackowski KM, Bastian AJ, van Zijl PC, Moser HW, Raymond GV, Golay X. Magnetization transfer MRI demonstrates spinal cord pathology in adrenomyeloneuropathy.Neurology 1739–1745, 2005. [DOI] [PubMed]
  • 41.Smith KD, Kemp S, Braiterman LT, Lu JF, Wei HM, Geraghty M, Stetten G, Bergin JS, Pevsner J, Watkins PA. X-linked adrenoleukodystrophy: genes, mutations, and phenotypes. Neurochem Res. 1999;24:521–535. doi: 10.1023/A:1022535930009. [DOI] [PubMed] [Google Scholar]
  • 42.van Geel BM, Bezman L, Loes DJ, Moser HW, Raymond GV. Evolution of phenotypes in adult male patients with X-linked adrenoleukodystrophy. Ann Neurol. 2001;49:186–94. doi: 10.1002/1531-8249(20010201)49:2<186::AID-ANA38>3.0.CO;2-R. [DOI] [PubMed] [Google Scholar]
  • 43.Aubourg P, Chaussain JL. Adrenoleukodystrophy: The most frequent genetic cause of Addison’s disease. Horm Res. 2003;59:104–105. doi: 10.1159/000067833. [DOI] [PubMed] [Google Scholar]
  • 44.Jorge P, Quelhas D, Oliveira P, Pinto R, Nogueira A. X-linked adrenoleukodystrophy in patients with idiopathic Addison disease. Eur J Pediatr. 1994;153:594–7. doi: 10.1007/BF02190668. [DOI] [PubMed] [Google Scholar]
  • 45.Dubey P, Raymond G, Moser AB, Kharkar S, Bezman L, Moser HW. Adrenal insufficiency in asymptomatic adrenoleukodystrophy patients identified by very long chain fatty acid screening. J Pediatr. 2005;146:528–532. doi: 10.1016/j.jpeds.2004.10.067. [DOI] [PubMed] [Google Scholar]
  • 46.Eichler FS, Barker PB, Cox C, Edwin D, Ulug AM, Moser HW, Raymond GV. Proton MR spectroscopic imaging predicts lesion progression on MRI in X-linked adrenoleukodystrophy. Neurology. 2002;58:901–907. doi: 10.1212/WNL.58.6.901. [DOI] [PubMed] [Google Scholar]
  • 47.Restuccia D, Di Lazzaro V, Valeriani M, Oliviero A, Le Pera D, Colosimo C, Burdi N, Cappa M, Bertini E, Di Biase A, Tonali P. Neurophysiological abnormalities in adrenoleukodystrophy carriers. Evidence of different degrees of central nervous system involvement. Brain. 1997;120:1139–1148. doi: 10.1093/brain/120.7.1139. [DOI] [PubMed] [Google Scholar]
  • 48.Fatemi A, Barker PB, Ulug AM, Nagae-Poetscher LM, Beauchamp NJ, Moser AB, Raymond GV, Moser HW, Naidu S. MRI and proton MRSI in women heterozygous for X-linked adrenoleukodystrophy. Neurology. 2003;60:1301–1307. doi: 10.1212/01.WNL.0000059546.15529.CB. [DOI] [PubMed] [Google Scholar]
  • 49.Hershkovitz E, Narkis G, Shorer Z, Moser AB, Watkins PA, Moser HW, Manor E. Cerebral X-linked adrenoleukodystrophy in a girl with Xq27-Ter deletion. Ann Neurol. 2002;52:234–237. doi: 10.1002/ana.10248. [DOI] [PubMed] [Google Scholar]
  • 50.Naidu S, Washington C, Thirumalai S, Smith KD, Moser HW, Watkins PA. X-chromosome inactivation in symptomatic heterozygotes of X-linked adrenoleukodystrophy. Ann Neurol. 1997;42:498a–498a. doi: 10.1002/ana.410420104. [DOI] [Google Scholar]
  • 51.Maier EM, Kammerer S, Muntau AC, Wichers M, Braun A, Roscher AA. Symptoms in carriers of adrenoleukodystrophy relate to skewed X inactivation. Ann Neurol. 2002;52:683–688. doi: 10.1002/ana.10376. [DOI] [PubMed] [Google Scholar]
  • 52.Kemp S, Wei HM, Lu JF, Braiterman LT, McGuinness MC, Moser AB, Watkins PA, Smith KD. Gene redundancy and pharmacological gene therapy: implications for X-linked adrenoleukodystrophy. Nat Med. 1998;4:1261–1268. doi: 10.1038/3242. [DOI] [PubMed] [Google Scholar]
  • 53.McGovern MM, Wasserstein MP, Aron A, Perrine SP. Biochemical effect of intravenous arginine butyrate in X-linked adrenoleukodystrophy. J Pediatr. 2003;142:709–13. doi: 10.1067/mpd.2003.201. [DOI] [PubMed] [Google Scholar]
  • 54.Pai GS, Khan M, Barbosa E, Key LL, Craver JR, Cure JK, Betros R, Singh I. Lovastatin therapy for X-linked adrenoleukodystrophy: clinical and biochemical observations on 12 patients. Mol Genet Metab. 2000;69:312–322. doi: 10.1006/mgme.2000.2977. [DOI] [PubMed] [Google Scholar]
  • 55.Fourcade S, Savary S, Gondcaille C, Berger J, Netik A, Cadepond F, El Etr M, Molzer B, Bugaut M. Thyroid hormone induction of the adrenoleukodystrophy-related gene (ABCD2) Mol Pharmacol. 2003;63:1296–1303. doi: 10.1124/mol.63.6.1296. [DOI] [PubMed] [Google Scholar]
  • 56.Benhamida S, Pflumio F, Dubart-Kupperschmitt A, Zhao-Emonet JC, Cavazzana-Calvo M, Rocchiccioli F, Fichelson S, Aubourg P, Chameau P, Cartier N. Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice. Mol Ther. 2003;7:317–324. doi: 10.1016/S1525-0016(03)00002-9. [DOI] [PubMed] [Google Scholar]
  • 57.Zhang LX, Bakshi R, Fine E, Moser HW. Clinical and electro-physiological improvement of adrenomyeloneuropathy with steroid treatment. J Neurol Neurosurg Psychiatry. 2003;74:822–823. doi: 10.1136/jnnp.74.6.822. [DOI] [PMC free article] [PubMed] [Google Scholar]
  • 58.Rizzo WB, Leshner RT, Odone A, Dammann AL, Craft DA, Jensen ME, Jennings SS, Davis S, Jaitly R, Sgro JA. Dietary erucic acid therapy for X-linked adrenoleukodystrophy. Neurology. 1989;39:1415–1422. doi: 10.1212/WNL.39.11.1415. [DOI] [PubMed] [Google Scholar]
  • 59.van Geel BM, Assies J, Haverkort EB, Koelman JH, Verbeeten B, Wanders RJ, Barth PG. Progression of abnormalities in adrenomyeloneuropathy and neurologically asymptomatic X-linked adrenoleukodystrophy despite treatment with “Lorenzo’s oil”. J Neurol Neurosurg Psychiatry. 1999;67:290–299. doi: 10.1136/jnnp.67.3.290. [DOI] [PMC free article] [PubMed] [Google Scholar]
  • 60.Uziel G, Bertini E, Bardelli P, Rimoldi M, Gambetti M. Experience on therapy of adrenoleukodystrophy and adrenomyeloneuropathy. Dev Neurosci. 1991;13:274–279. doi: 10.1159/000112173. [DOI] [PubMed] [Google Scholar]
  • 61.Aubourg P, Adamsbaum C, Lavallard-Rousseau MC, Rocchiccioli F, Cartier N, Jambaque I, Jakobezak C, Lemaitre A, Boureau F, Wolf C, et al. A two-year trial of oleic and erucic acids (“Lorenzo’s oil”) as treatment for adrenomyeloneuropathy. N Engl J Med. 1993;329:745–752. doi: 10.1056/NEJM199309093291101. [DOI] [PubMed] [Google Scholar]
  • 62.Rizzo WB, Leshner RT, Odone A, Craft DA, Jennings SS, Jaitly R, et al. X-linked adrenoleukodystrophy: Biochemical and clinical efficacy of dietary erucic acid therapy. In: Uziel G, Wanders RJA, Cappa ME, et al., editors. Adrenoleukodystrophy and Other Peroxisomal Disorders. Amsterdam: Excerpta Medica; 1990. pp. 149–162. [Google Scholar]
  • 63.Koehler W, Sokolowski P. Clinical phenotypes, diagnosis and treatment of adulthood X-linked adrenoleukodystrophy. In: Berger J, Stoeckler S, Koehler W, editors. Understanding and Treating Adrenoleukodystrophy: Present State and Future Perspectives. Verlag Heilbronn: SPS Verlagsgeseltschaft; 2005. pp. 28–60. [Google Scholar]
  • 64.Peters C, Charnas LR, Tan Y, Ziegler RS, Shapiro EG, DeFor T, Grewal SS, Orchard PJ, Abel SL, Goldman AI, Ramsay NK, Dusenbery KE, Loes DJ, Lockman LA, Kato S, Aubourg PR, Moser HW, Krivit W. Cerebral X-linked adrenoleukodystrophy: The international hematopoietic cell transplantation experience from 1982 to 1999. Blood. 2004;104:881–888. doi: 10.1182/blood-2003-10-3402. [DOI] [PubMed] [Google Scholar]
  • 65.Aubourg P, Blanche S, Jambaque I, Rocchiccioli F, Kalifa G, Naud-Saudreau C, Rolland MO, Debre M, Chaussain JL, Griscelli C, et al. Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation. N Engl J Med. 1990;322:1860–1866. doi: 10.1056/NEJM199006283222607. [DOI] [PubMed] [Google Scholar]
  • 66.Shapiro E, Krivit W, Lockman L, Jambaque I, Peters C, Cowan M, Harris R, Blanche S, Bordigoni P, Loes D, Ziegler R, Crittenden M, Ris D, Berg B, Cox C, Moser H, Fischer A, Aubourg P. Long-term effect of bone marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy. Lancet. 2000;356:713–718. doi: 10.1016/S0140-6736(00)02629-5. [DOI] [PubMed] [Google Scholar]
  • 67.Hickey WF, Kimura H. Perivascular microglial cells of the CNS are bone marrow-derived and present antigen in vivo. Science. 1988;239:290–292. doi: 10.1126/science.3276004. [DOI] [PubMed] [Google Scholar]
  • 68.Unger ER, Sung JH, Manivel JC, Chenggis ML, Blazar BR, Krivit W. Male donor-derived cells in the brains of female sex-mismatched bone marrow transplant recipients: a Y-chromosome specific in situ hybridization study. J Neuropathol Exp Neurol. 1993;52:460–470. doi: 10.1097/00005072-199309000-00004. [DOI] [PubMed] [Google Scholar]
  • 69.Nowaczyk MJ, Saunders EF, Tein I, Blaser SI, Clarke JT. Immunoablation does not delay the neurologic progression of X-linked adrenoleukodystrophy. J Pediatr. 1997;131:453–455. doi: 10.1016/S0022-3476(97)80076-5. [DOI] [PubMed] [Google Scholar]
  • 70.Mahmood A, Dubey P, Raymond GV, Moser HW. Evaluating survival in nontransplanted boys with cerebral phenotype of X-linked adrenoleukodystrophy.Ann Neurol in press, 2005.
  • 71.Maier EM, Roscher AA, Kammerer S, Mehnert K, Conzelmann E, Holzinger A. Prenatal diagnosis of X-linked adrenoleukodystrophy combining biochemical, immunocytochemical and DNA analyses. Prenat Diagn. 1999;19:364–8. doi: 10.1002/(SICI)1097-0223(199904)19:4<364::AID-PD525>3.0.CO;2-W. [DOI] [PubMed] [Google Scholar]

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