Literature DB >> 23647506

Allogeneic hematopoietic stem cell transplantation for treatment of Gaucher disease.

Maciej Machaczka.   

Abstract

Ito and Barrett recently reviewed the international experiences with allogeneic hematopoietic stem cell transplantation (allo-HSCT) for the treatment of the neuronopathic and non-neuronopathic forms of Gaucher disease (GD). In the present communication, the author adds some updated data on the long-term outcome of allo-HSCT for Swedish GD patients with the mean and median follow-up time of 27 years (the longest follow-up of 30 years) and makes comments on some of the authors' statements found in the article.

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Year:  2013        PMID: 23647506     DOI: 10.3109/08880018.2013.793757

Source DB:  PubMed          Journal:  Pediatr Hematol Oncol        ISSN: 0888-0018            Impact factor:   1.969


  5 in total

Review 1.  The clinical management of Type 2 Gaucher disease.

Authors:  Karin Weiss; Ashley Gonzalez; Grisel Lopez; Leah Pedoeim; Catherine Groden; Ellen Sidransky
Journal:  Mol Genet Metab       Date:  2014-11-14       Impact factor: 4.797

Review 2.  Treatment of lysosomal storage disorders: successes and challenges.

Authors:  Carla E M Hollak; Frits A Wijburg
Journal:  J Inherit Metab Dis       Date:  2014-05-13       Impact factor: 4.982

3.  Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing.

Authors:  Samantha G Scharenberg; Edina Poletto; Katherine L Lucot; Pasqualina Colella; Adam Sheikali; Thomas J Montine; Matthew H Porteus; Natalia Gomez-Ospina
Journal:  Nat Commun       Date:  2020-07-03       Impact factor: 14.919

4.  Neurocognitive profile of adults with the Norrbottnian type of Gaucher disease.

Authors:  Panagiota Tsitsi; Ioanna Markaki; Josefine Waldthaler; Maciej Machaczka; Per Svenningsson
Journal:  JIMD Rep       Date:  2021-11-21

5.  CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.

Authors:  John Marshall; Ying Sun; Dinesh S Bangari; Eva Budman; Hyejung Park; Jennifer B Nietupski; Amy Allaire; Mary A Cromwell; Bing Wang; Gregory A Grabowski; John P Leonard; Seng H Cheng
Journal:  Mol Ther       Date:  2016-03-07       Impact factor: 11.454

  5 in total

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