Scoring Evaluation of the Natural Course of Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Sanfilippo Syndrome ◽

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 ◽

Natural history of siblings with mucopolysaccharidosis type IIIA, Sanfilippo syndrome type A

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2016.11.127 ◽

2017 ◽

Vol 120 (1-2) ◽

pp. S57-S58

Author(s):

Alexandra Gossler ◽

Anja Koehn ◽

Ann Meyer ◽

Kai Kossow ◽

Kurt Ullrich ◽

...

Keyword(s):

Natural History ◽

Type A ◽

Syndrome Type ◽

Sanfilippo Syndrome ◽

Type Iiia ◽

History Of ◽

Identification and characterization of mutations underlying Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA)

Clinical Genetics ◽

10.1034/j.1399-0004.2002.610304.x ◽

2002 ◽

Vol 61 (3) ◽

pp. 192-197 ◽

Cited By ~ 14

Author(s):

GJ Lee-Chen ◽

SP Lin ◽

MH Ko ◽

CK Chuang ◽

CP Chen ◽

...

Keyword(s):

Type A ◽

Syndrome Type ◽

Sanfilippo Syndrome ◽

Type Iiia ◽

Identification And Characterization ◽

A Multiparametric Computational Algorithm for Comprehensive Assessment of Genetic Mutations in Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)

PLoS ONE ◽

10.1371/journal.pone.0121511 ◽

2015 ◽

Vol 10 (3) ◽

pp. e0121511 ◽

Cited By ~ 5

Author(s):

Krastyu G. Ugrinov ◽

Stefan D. Freed ◽

Clayton L. Thomas ◽

Shaun W. Lee

Keyword(s):

Computational Algorithm ◽

Comprehensive Assessment ◽

Genetic Mutations ◽

Sanfilippo Syndrome ◽

Type Iiia ◽

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 ◽

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

Experimental Neurology ◽

10.1016/j.expneurol.2018.01.020 ◽

2018 ◽

Vol 303 ◽

pp. 38-47 ◽

Cited By ~ 7

Author(s):

Dani L. Webber ◽

Amanda Choo ◽

Laura J. Hewson ◽

Paul J. Trim ◽

Marten F. Snel ◽

...

Keyword(s):

Heparan Sulfate ◽

Pathogenic Mechanisms ◽

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 ◽