Literature DB >> 2863494

Bone-marrow transplantation for metachromatic leucodystrophy.

E Bayever, S Ladisch, M Philippart, N Brill, M Nuwer, R S Sparkes, S A Feig.   

Abstract

An 11-month-old boy with late infantile metachromatic leucodystrophy was given a bone-marrow transplant (BMT) from an HLA-identical sister; 6 months later his cerebrospinal fluid leucocytes were exclusively of donor origin. Coupled with the patient's continued developmental progress, as assessed 33 months after the procedure, the findings suggest that BMT may be an effective treatment for some congenital metabolic disorders which affect the central nervous system.

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Year:  1985        PMID: 2863494     DOI: 10.1016/s0140-6736(85)90402-7

Source DB:  PubMed          Journal:  Lancet        ISSN: 0140-6736            Impact factor:   79.321


  15 in total

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Review 2.  Enzyme-replacement therapy: problems and prospects.

Authors:  B Rademaker; J Raber
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3.  Unrelated umbilical cord blood transplant for juvenile metachromatic leukodystrophy: a 5-year follow-up in three affected siblings.

Authors:  Casey Cable; Richard S Finkel; Tanya J Lehky; Nadia M Biassou; Edythe A Wiggs; Nancy Bunin; Tyler Mark Pierson
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4.  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

5.  Bone marrow transplantation in the treatment of alpha-mannosidosis.

Authors:  A Will; A Cooper; C Hatton; I B Sardharwalla; D I Evans; R F Stevens
Journal:  Arch Dis Child       Date:  1987-10       Impact factor: 3.791

Review 6.  Current Perspective of Stem Cell Therapy in Neurodegenerative and Metabolic Diseases.

Authors:  Ajay Kumar; Karthikeyan Narayanan; Ravi Kumar Chaudhary; Sachin Mishra; Sundramurthy Kumar; Kumar Jayaseelan Vinoth; Parasuraman Padmanabhan; Balázs Gulyás
Journal:  Mol Neurobiol       Date:  2016-11-04       Impact factor: 5.590

7.  Secretion of phosphomannosyl-deficient arylsulphatase A and cathepsin D from isolated human macrophages.

Authors:  Nicole Muschol; Ulrich Matzner; Stephan Tiede; Volkmar Gieselmann; Kurt Ullrich; Thomas Braulke
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8.  Haematopoietic stem cell transplantation does not retard disease progression in the psycho-cognitive variant of late-onset metachromatic leukodystrophy.

Authors:  Nicholas J Smith; Robert E Marcus; Barbara J Sahakian; Narinder Kapur; Timothy M Cox
Journal:  J Inherit Metab Dis       Date:  2010-11-16       Impact factor: 4.982

9.  Hematopoietic cell transplantation in murine globoid cell leukodystrophy (the twitcher mouse): effects on levels of galactosylceramidase, psychosine, and galactocerebrosides.

Authors:  T Ichioka; Y Kishimoto; S Brennan; G W Santos; A M Yeager
Journal:  Proc Natl Acad Sci U S A       Date:  1987-06       Impact factor: 11.205

10.  Effect of bone marrow transplantation on enzyme levels and clinical course in the neurologically affected twitcher mouse.

Authors:  P M Hoogerbrugge; B J Poorthuis; A E Romme; J J van de Kamp; G Wagemaker; D W van Bekkum
Journal:  J Clin Invest       Date:  1988-06       Impact factor: 14.808

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