Literature DB >> 17237829

Early marrow transplantation in a pre-symptomatic neonate with late infantile metachromatic leukodystrophy does not halt disease progression.

R G M Bredius, L A E M Laan, A C Lankester, B J H M Poorthuis, M J D van Tol, R M Egeler, W F M Arts.   

Abstract

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Year:  2007        PMID： 17237829     DOI： 10.1038/sj.bmt.1705581

Source DB:  PubMed          Journal:  Bone Marrow Transplant        ISSN： 0268-3369            Impact factor:   5.483


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  11 in total

1.  Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.

Authors:  Françoise Piguet; Dolan Sondhi; Monique Piraud; Françoise Fouquet; Neil R Hackett; Ornella Ahouansou; Marie-Thérèse Vanier; Ivan Bieche; Patrick Aubourg; Ronald G Crystal; Nathalie Cartier; Caroline Sevin
Journal:  Hum Gene Ther       Date:  2012-07-23       Impact factor: 5.695

2.  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

3.  Disease specific therapies in leukodystrophies and leukoencephalopathies.

Authors:  Guy Helman; Keith Van Haren; Joshua L Bonkowsky; Genevieve Bernard; Amy Pizzino; Nancy Braverman; Dean Suhr; Marc C Patterson; S Ali Fatemi; Jeff Leonard; Marjo S van der Knaap; Stephen A Back; Stephen Damiani; Steven A Goldman; Asako Takanohashi; Magdalena Petryniak; David Rowitch; Albee Messing; Lawrence Wrabetz; Raphael Schiffmann; Florian Eichler; Maria L Escolar; Adeline Vanderver
Journal:  Mol Genet Metab       Date:  2015-02-07       Impact factor: 4.797

Review 4.  Emerging treatments for pediatric leukodystrophies.

Authors:  Guy Helman; Keith Van Haren; Maria L Escolar; Adeline Vanderver
Journal:  Pediatr Clin North Am       Date:  2015-04-08       Impact factor: 3.278

Review 5.  Adulthood leukodystrophies.

Authors:  Wolfgang Köhler; Julian Curiel; Adeline Vanderver
Journal:  Nat Rev Neurol       Date:  2018-01-05       Impact factor: 42.937

6.  Advances in the Treatment of Neuronal Ceroid Lipofuscinosis.

Authors:  Jonathan B Rosenberg; Alvin Chen; Stephen M Kaminsky; Ronald G Crystal; Dolan Sondhi
Journal:  Expert Opin Orphan Drugs       Date:  2019-11-27       Impact factor: 0.694

Review 7.  Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism.

Authors:  Emily Y Tan; Jaap Jan Boelens; Simon A Jones; Robert F Wynn
Journal:  Front Pediatr       Date:  2019-10-25       Impact factor: 3.418

8.  Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report.

Authors:  Alexander A Boucher; Weston Miller; Ryan Shanley; Richard Ziegler; Troy Lund; Gerald Raymond; Paul J Orchard
Journal:  Orphanet J Rare Dis       Date:  2015-08-07       Impact factor: 4.123

Review 9.  Developing therapeutic approaches for metachromatic leukodystrophy.

Authors:  Shilpa A Patil; Gustavo H B Maegawa
Journal:  Drug Des Devel Ther       Date:  2013-08-08       Impact factor: 4.162

Review 10.  Metachromatic Leukodystrophy: Diagnosis, Modeling, and Treatment Approaches.

Authors:  Alisa A Shaimardanova; Daria S Chulpanova; Valeriya V Solovyeva; Aysilu I Mullagulova; Kristina V Kitaeva; Cinzia Allegrucci; Albert A Rizvanov
Journal:  Front Med (Lausanne)       Date:  2020-10-20
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