Literature DB >> 16151914

Bone marrow transplantation correcting beta-galactosidase activity does not influence neurological outcome in juvenile GM1-gangliosidosis.

J P H Shield1, J Stone, C G Steward.   

Abstract

Donor bone marrow engraftment, which resulted in complete normalization of white cell beta-galactosidase levels in a patient with presymptomatic juvenile onset GM1-gangliosidosis (McKusick 230600), did not improve long-term clinical outcome.

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Year:  2005        PMID： 16151914     DOI： 10.1007/s10545-005-0089-7

Source DB:  PubMed          Journal:  J Inherit Metab Dis        ISSN： 0141-8955            Impact factor:   4.982


  3 in total

1.  Bone marrow stem cells have the ability to populate the entire central nervous system into fully differentiated parenchymal microglia.

Authors:  Alain R Simard; Serge Rivest
Journal:  FASEB J       Date:  2004-04-14       Impact factor: 5.191

2.  Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group.

Authors:  C Peters; E G Shapiro; J Anderson; P J Henslee-Downey; M R Klemperer; M J Cowan; E F Saunders; P A deAlarcon; C Twist; J B Nachman; G A Hale; R E Harris; M K Rozans; J Kurtzberg; G H Grayson; T E Williams; C Lenarsky; J E Wagner; W Krivit
Journal:  Blood       Date:  1998-04-01       Impact factor: 22.113

3.  Bone marrow transplantation in canine GM1 gangliosidosis.

Authors:  J S O'Brien; R Storb; R F Raff; J Harding; F Appelbaum; S Morimoto; Y Kishimoto; T Graham; A Ahern-Rindell; S L O'Brien
Journal:  Clin Genet       Date:  1990-10       Impact factor: 4.438
  3 in total
  11 in total

Review 1.  The GM1 and GM2 Gangliosidoses: Natural History and Progress toward Therapy.

Authors:  Debra S Regier; Richard L Proia; Alessandra D'Azzo; Cynthia J Tifft
Journal:  Pediatr Endocrinol Rev       Date:  2016-06

2.  Child neurology: Exaggerated dermal melanocytosis in a hypotonic infant: a harbinger of GM1 gangliosidosis.

Authors:  Amy Armstrong-Javors; Catherine J Chu
Journal:  Neurology       Date:  2014-10-21       Impact factor: 9.910

Review 3.  Potential use of stem cells as a therapy for cystinosis.

Authors:  Celine J Rocca; Stephanie Cherqui
Journal:  Pediatr Nephrol       Date:  2018-05-22       Impact factor: 3.714

4.  Haematopoietic Stem Cell Transplantation Arrests the Progression of Neurodegenerative Disease in Late-Onset Tay-Sachs Disease.

Authors:  Karolina M Stepien; Su Han Lum; J Edmond Wraith; Christian J Hendriksz; Heather J Church; David Priestman; Frances M Platt; Simon Jones; Ana Jovanovic; Robert Wynn
Journal:  JIMD Rep       Date:  2017-12-07

5.  Gene therapy for GM1 gangliosidosis: challenges of translational medicine.

Authors:  Carl Hayward; Hitesh C Patel; Sanjay G Manohar; Alexander R Lyon
Journal:  Ann Transl Med       Date:  2015-05

6.  Fast urinary screening of oligosaccharidoses by MALDI-TOF/TOF mass spectrometry.

Authors:  Laurent Bonesso; Monique Piraud; Céline Caruba; Emmanuel Van Obberghen; Raymond Mengual; Charlotte Hinault
Journal:  Orphanet J Rare Dis       Date:  2014-02-06       Impact factor: 4.123

7.  Novel Drug Candidates Improve Ganglioside Accumulation and Neural Dysfunction in GM1 Gangliosidosis Models with Autophagy Activation.

Authors:  Ryutaro Kajihara; Tadahiro Numakawa; Haruki Odaka; Yuji Yaginuma; Noemi Fusaki; Toshika Okumiya; Hirokazu Furuya; Seiji Inui; Takumi Era
Journal:  Stem Cell Reports       Date:  2020-04-16       Impact factor: 7.765

8.  Intracellular Delivery of β-Galactosidase Enzyme Using Arginase-Responsive Dextran Sulfate/Poly-l-arginine Capsule for Lysosomal Storage Disorder.

Authors:  Meenakshi Gupta; Himanshu Pandey; Sri Sivakumar
Journal:  ACS Omega       Date:  2017-12-15

9.  Axonopathy and Reduction of Membrane Resistance: Key Features in a New Murine Model of Human GM1-Gangliosidosis.

Authors:  Deborah Eikelberg; Annika Lehmbecker; Graham Brogden; Witchaya Tongtako; Kerstin Hahn; Andre Habierski; Julia B Hennermann; Hassan Y Naim; Felix Felmy; Wolfgang Baumgärtner; Ingo Gerhauser
Journal:  J Clin Med       Date:  2020-04-02       Impact factor: 4.241

10.  A Single Injection of an Optimized Adeno-Associated Viral Vector into Cerebrospinal Fluid Corrects Neurological Disease in a Murine Model of GM1 Gangliosidosis.

Authors:  Christian Hinderer; Brenden Nosratbakhsh; Nathan Katz; James M Wilson
Journal:  Hum Gene Ther       Date:  2020-11-02       Impact factor: 5.695
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