Systemic scAAV9.U1a.hSGSH delivery corrects brain biochemistry in mucopolysaccharidosis type IIIA at early and later stages of disease

2020 ◽  
Author(s):  
Jennifer Saville ◽  
Ainslie Derrick-Roberts ◽  
Chantelle McIntyre ◽  
Maria Fuller
Keyword(s):  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

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

2014 ◽  
Vol 1 ◽  
pp. 422-424 ◽  
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 ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

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

2014 ◽  
Vol 16 (11-12) ◽  
pp. 374-387 ◽  
Author(s):  
Chantelle McIntyre ◽  
Ainslie L. K. Derrick-Roberts ◽  
Sharon Byers ◽  
Donald S. Anson
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Gene Delivery ◽  
Mucopolysaccharidosis Type ◽  
Central Nervous System Pathology ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

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

2018 ◽  
Vol 303 ◽  
pp. 38-47 ◽  
Author(s):  
Dani L. Webber ◽  
Amanda Choo ◽  
Laura J. Hewson ◽  
Paul J. Trim ◽  
Marten F. Snel ◽  
...  
Keyword(s):  
Heparan Sulfate ◽  
Mucopolysaccharidosis Type ◽  
Pathogenic Mechanisms ◽  
Type Iiia ◽  
Mucopolysaccharidosis 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)

2020 ◽  
Vol 129 (2) ◽  
pp. S56-S57 ◽  
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 ◽  
Mucopolysaccharidosis Type ◽  
Sanfilippo Syndrome ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

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

2019 ◽  
Vol 126 (2) ◽  
pp. S111-S112
Author(s):  
Sophie Olivier ◽  
Samantha Parker ◽  
Anupam Chakrapani ◽  
Ronald Crystal ◽  
Lisa Emrick ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Mps Iiia ◽  
Mucopolysaccharidosis Type Iiia

scholarly journals Pentosan Polysulfate Treatment of Mucopolysaccharidosis Type IIIA Mice

2018 ◽  
pp. 37-52 ◽  
Author(s):  
Ningning Guo ◽  
Victor DeAngelis ◽  
Changzhi Zhu ◽  
Edward H. Schuchman ◽  
Calogera M. Simonaro
Keyword(s):  
Mucopolysaccharidosis Type ◽  
Pentosan Polysulfate ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

scholarly journals A novel mutation of SGSH and clinical features analysis of mucopolysaccharidosis type IIIA

Medicine ◽  
2018 ◽  
Vol 97 (52) ◽  
pp. e13758 ◽  
Author(s):  
Xiaohua Li ◽  
Rui Xiao ◽  
Baiyu Chen ◽  
Guanglu Yang ◽  
Xiaomeng Zhang ◽  
...  
Keyword(s):  
Clinical Features ◽  
Novel Mutation ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

Lentiviral mediated stem cell gene therapy corrects a mouse model of mucopolysaccharidosis type IIIA

2011 ◽  
Vol 102 (2) ◽  
pp. S8
Author(s):  
Brian Bigger ◽  
Alex Langford-Smith ◽  
Fiona Wilkinson ◽  
Kia Langford-Smith ◽  
Ana Sergijenko ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Mouse Model ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Cell Gene ◽  
Mucopolysaccharidosis Type Iiia ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy
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