Literature DB >> 10546790

Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome.

W Krivit1, P Aubourg, E Shapiro, C Peters.   

Abstract

Bone marrow transplantation protocols for inherited metabolic storage diseases are unique for each disorder treated. Differences depend also upon how old the patient was when onset occurred and rate of progression of disease. Treatment is directed to prevent or ameliorate the inexorable neurological deterioration that is the major pathophysiological event in all of these inherited metabolic storage diseases.

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Mesh：

Year:  1999        PMID： 10546790     DOI： 10.1097/00062752-199911000-00004

Source DB:  PubMed          Journal:  Curr Opin Hematol        ISSN： 1065-6251            Impact factor:   3.284


  30 in total

1.  Early diagnosis of cerebral X-linked adrenoleukodystrophy in boys with Addison's disease improves survival and neurological outcomes.

Authors:  Lynda E Polgreen; Saydi Chahla; Weston Miller; Steven Rothman; Jakub Tolar; Teresa Kivisto; David Nascene; Paul J Orchard; Anna Petryk
Journal:  Eur J Pediatr       Date:  2011-01-29       Impact factor: 3.183

2.  Co-receptor and co-stimulation blockade for mixed chimerism and tolerance without myelosuppressive conditioning.

Authors:  Luis Graca; Stephen Daley; Paul J Fairchild; Stephen P Cobbold; Herman Waldmann
Journal:  BMC Immunol       Date:  2006-04-25       Impact factor: 3.615

Review 3.  Gene therapy for the neurological manifestations in lysosomal storage disorders.

Authors:  Seng H Cheng
Journal:  J Lipid Res       Date:  2014-03-29       Impact factor: 5.922

Review 4.  Gene therapy for metachromatic leukodystrophy.

Authors:  Jonathan B Rosenberg; Stephen M Kaminsky; Patrick Aubourg; Ronald G Crystal; Dolan Sondhi
Journal:  J Neurosci Res       Date:  2016-11       Impact factor: 4.164

5.  Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates.

Authors:  Jonathan B Rosenberg; Dolan Sondhi; David G Rubin; Sébastien Monette; Alvin Chen; Sara Cram; Bishnu P De; Stephen M Kaminsky; Caroline Sevin; Patrick Aubourg; Ronald G Crystal
Journal:  Hum Gene Ther Clin Dev       Date:  2014-08-21       Impact factor: 5.032

6.  The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse.

Authors:  Dan Wang; Valery Belakhov; Jeyakumar Kandasamy; Timor Baasov; Su-Chen Li; Yu-Teh Li; David M Bedwell; Kim M Keeling
Journal:  Mol Genet Metab       Date:  2011-10-19       Impact factor: 4.797

Review 7.  Transplantation as disease modifying therapy in adults with inherited metabolic disorders.

Authors:  Sandra Sirrs; Fady Hannah-Shmouni; Stephen Nantel; James Neuberger; Eric M Yoshida
Journal:  J Inherit Metab Dis       Date:  2018-02-01       Impact factor: 4.982

Review 8.  Gene therapy for lysosomal storage diseases (LSDs) in large animal models.

Authors:  Mark Haskins
Journal:  ILAR J       Date:  2009

9.  Tandem mass spectrometry for the direct assay of lysosomal enzymes in dried blood spots: application to screening newborns for mucopolysaccharidosis I.

Authors:  Sophie Blanchard; Martin Sadilek; C Ronald Scott; Frantisek Turecek; Michael H Gelb
Journal:  Clin Chem       Date:  2008-12       Impact factor: 8.327

10.  Unrelated allogeneic bone marrow transplant in adrenoleukodystrophy using CD34+ stem cell selection.

Authors:  Anupama Borker; Lolie C Yu
Journal:  Metab Brain Dis       Date:  2002-09       Impact factor: 3.584
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