Literature DB >> 14722533

Successful hematopoietic stem cell transplantation in Farber disease.

Josef Vormoor1, Karoline Ehlert, Andreas H Groll, Hans-Georg Koch, Michael Frosch, Johannes Roth.   

Abstract

Farber disease, a lysosomal storage disorder, has a dismal prognosis leading to death with progressive granulomatous inflammation, even in patients without central nervous system involvement (type 2/3). We report the first successful hematopoietic stem cell transplantations in 2 patients with Farber disease type 2/3, resulting in resolution of symptoms.

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Year:  2004        PMID： 14722533     DOI： 10.1016/j.jpeds.2003.09.051

Source DB:  PubMed          Journal:  J Pediatr        ISSN： 0022-3476            Impact factor:   4.406


  9 in total

1.  Farber's disease without central nervous system involvement: bone-marrow transplantation provides a promising new approach.

Authors:  K Ehlert; J Roth; M Frosch; N Fehse; N Zander; J Vormoor
Journal:  Ann Rheum Dis       Date:  2006-12       Impact factor: 19.103

2.  Autologous transplantation of lentivector/acid ceramidase-transduced hematopoietic cells in nonhuman primates.

Authors:  Jagdeep S Walia; Anton Neschadim; Orlay Lopez-Perez; Abdulfatah Alayoubi; Xin Fan; Stéphane Carpentier; Melissa Madden; Chyan-Jang Lee; Fred Cheung; David A Jaffray; Thierry Levade; J Andrea McCart; Jeffrey A Medin
Journal:  Hum Gene Ther       Date:  2011-03-25       Impact factor: 5.695

3.  In vivo delivery of human acid ceramidase via cord blood transplantation and direct injection of lentivirus as novel treatment approaches for Farber disease.

Authors:  Shobha Ramsubir; Takahiro Nonaka; Carmen Bedia Girbés; Stéphane Carpentier; Thierry Levade; Jeffrey A Medin
Journal:  Mol Genet Metab       Date:  2008-09-20       Impact factor: 4.797

4.  Odontoid infiltration and spinal compression in Farber Disease: reversal by haematopoietic stem cell transplantation.

Authors:  Andrea Jarisch; Colin G Steward; Jan Sörensen; Luciana Porto; Matthias Kieslich; Thomas Klingebiel; Peter Bader
Journal:  Eur J Pediatr       Date:  2013-07-24       Impact factor: 3.183

5.  Farber disease: clinical presentation, pathogenesis and a new approach to treatment.

Authors:  Karoline Ehlert; Michael Frosch; Natalja Fehse; Axel Zander; Johannes Roth; Josef Vormoor
Journal:  Pediatr Rheumatol Online J       Date:  2007-06-29       Impact factor: 3.054

Review 6.  Acid ceramidase deficiency: Farber disease and SMA-PME.

Authors:  Fabian P S Yu; Samuel Amintas; Thierry Levade; Jeffrey A Medin
Journal:  Orphanet J Rare Dis       Date:  2018-07-20       Impact factor: 4.123

Review 7.  Stem Cell Research Tools in Human Metabolic Disorders: An Overview.

Authors:  Serena Ricci; Pietro Cacialli
Journal:  Cells       Date:  2021-10-07       Impact factor: 6.600

8.  Farber Disease Mimicking Juvenile Idiopathic Arthritis: The First Reported Case in Qatar and Review of the Literature.

Authors:  Amal Al-Naimi; Haneen Toma; Sara G Hamad; Tawfeg Ben Omran
Journal:  Case Rep Genet       Date:  2022-02-10

9.  Deletion of MCP-1 Impedes Pathogenesis of Acid Ceramidase Deficiency.

Authors:  Fabian P S Yu; Shaalee Dworski; Jeffrey A Medin
Journal:  Sci Rep       Date:  2018-01-29       Impact factor: 4.379
  9 in total

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