Neuronal-specific impairment of heparan sulfate degradation in Drosophila reveals pathogenic mechanisms for Mucopolysaccharidosis type IIIA

Pathogenic Mechanisms ◽

Type Iiia ◽

Validation of a heparan sulfate-derived disaccharide as a marker of accumulation in murine mucopolysaccharidosis type IIIA

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2005.09.026 ◽

2006 ◽

Vol 87 (2) ◽

pp. 107-112 ◽

Cited By ~ 28

Author(s):

B KING ◽

P SAVAS ◽

M FULLER ◽

J HOPWOOD ◽

K HEMSLEY

Keyword(s):

Heparan Sulfate ◽

Type Iiia ◽

Mild mental retardation and low levels of urinary heparan sulfate in a patient with the attenuated phenotype of mucopolysaccharidosis type IIIA

Clinical Biochemistry ◽

10.1016/j.clinbiochem.2012.12.014 ◽

2013 ◽

Vol 46 (7-8) ◽

pp. 688-690 ◽

Cited By ~ 5

Author(s):

Giovanni V. Coppa ◽

Fabio Galeotti ◽

Lucia Zampini ◽

Tiziana Galeazzi ◽

Lucia Padella ◽

...

Keyword(s):

Mental Retardation ◽

Heparan Sulfate ◽

Mild Mental Retardation ◽

Type Iiia ◽

Low Levels ◽

Reduction in Brain Heparan Sulfate with Systemic Administration of an IgG Trojan Horse–Sulfamidase Fusion Protein in the Mucopolysaccharidosis Type IIIA Mouse

Molecular Pharmaceutics ◽

10.1021/acs.molpharmaceut.7b00958 ◽

2017 ◽

Vol 15 (2) ◽

pp. 602-608 ◽

Cited By ~ 11

Author(s):

Ruben J. Boado ◽

Jeff Zhiqiang Lu ◽

Eric Ka-Wai Hui ◽

William M. Pardridge

Keyword(s):

Fusion Protein ◽

Heparan Sulfate ◽

Trojan Horse ◽

Systemic Administration ◽

Type Iiia ◽

Distribution of Heparan Sulfate Oligosaccharides in Murine Mucopolysaccharidosis Type IIIA

Metabolites ◽

10.3390/metabo4041088 ◽

2014 ◽

Vol 4 (4) ◽

pp. 1088-1100 ◽

Cited By ~ 3

Author(s):

Kerryn Mason ◽

Peter Meikle ◽

John Hopwood ◽

Maria Fuller

Keyword(s):

Heparan Sulfate ◽

Type Iiia ◽

Heparan Sulfate Oligosaccharides ◽

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA

Case Medical Research ◽

10.31525/ct1-nct04201405 ◽

2019 ◽

Author(s):

Keyword(s):

Stem Cells ◽

Gene Therapy ◽

Hematopoietic Stem Cells ◽

Hematopoietic Stem ◽

Type Iiia ◽

Cardiac disease as the presenting feature of mucopolysaccharidosis type IIIA: A case report

Molecular Genetics and Metabolism Reports ◽

10.1016/j.ymgmr.2014.09.003 ◽

2014 ◽

Vol 1 ◽

pp. 422-424 ◽

Cited By ~ 5

Author(s):

Erlane Marques Ribeiro ◽

Ana Carolina Brusius-Facchin ◽

Sandra Leistner-Segal ◽

Carlos Antônio Bruno da Silva ◽

Ida Vanessa Schwartz

Keyword(s):

Case Report ◽

Cardiac Disease ◽

Type Iiia ◽

Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral-mediated gene delivery

The Journal of Gene Medicine ◽

10.1002/jgm.2816 ◽

2014 ◽

Vol 16 (11-12) ◽

pp. 374-387 ◽

Cited By ~ 10

Author(s):

Chantelle McIntyre ◽

Ainslie L. K. Derrick-Roberts ◽

Sharon Byers ◽

Donald S. Anson

Keyword(s):

Central Nervous System ◽

Nervous System ◽

Gene Delivery ◽

Central Nervous System Pathology ◽

Type Iiia ◽

Characterization of Sulfated Oligosaccharides in Mucopolysaccharidosis Type IIIA by Electrospray Ionization Mass Spectrometry

Analytical Chemistry ◽

10.1021/ac052083d ◽

2006 ◽

Vol 78 (13) ◽

pp. 4534-4542 ◽

Cited By ~ 28

Author(s):

Kerryn E. Mason ◽

Peter J. Meikle ◽

John J. Hopwood ◽

Maria Fuller

Keyword(s):

Mass Spectrometry ◽

Electrospray Ionization ◽

Electrospray Ionization Mass Spectrometry ◽

Ionization Mass Spectrometry ◽

Ionization Mass ◽

Sulfated Oligosaccharides ◽

Type Iiia ◽

Interim results of Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA)

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2019.11.129 ◽

2020 ◽

Vol 129 (2) ◽

pp. S56-S57 ◽

Cited By ~ 1

Author(s):

Kevin M. Flanigan ◽

Nicholas J.C. Smith ◽

Maria Luz Couce ◽

Kristen V. Truxal ◽

Kim L. McBride ◽

...

Keyword(s):

Clinical Trial ◽

Gene Therapy ◽

Single Dose ◽

Phase 1 ◽

Type A ◽

Syndrome Type ◽

Sanfilippo Syndrome ◽

Type Iiia ◽

Design and rationale of the LYS-SAF302 gene therapy study in mucopolysaccharidosis type IIIA (MPS IIIA) children

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2018.12.283 ◽

2019 ◽

Vol 126 (2) ◽

pp. S111-S112

Author(s):

Sophie Olivier ◽

Samantha Parker ◽

Anupam Chakrapani ◽

Ronald Crystal ◽

Lisa Emrick ◽

...

Keyword(s):

Gene Therapy ◽

Type Iiia ◽

Mps Iiia ◽