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

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

Pre-clinical workup of lentiviral mediated stem cell gene therapy for mucopolysaccharidosis type IIIA

2015 ◽  
Vol 114 (2) ◽  
pp. S41
Author(s):  
Stuart M. Ellison ◽  
Ana Sergijenko ◽  
Alex Langford-Smith ◽  
Kia Langford-Smith ◽  
Fiona Wilkinson ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Cell Gene ◽  
Mucopolysaccharidosis Type Iiia ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Early disease course is unaltered in mucopolysaccharidosis type IIIA (MPS IIIA) mice lacking α‐synuclein

2019 ◽  
Vol 45 (7) ◽  
pp. 715-731 ◽  
Author(s):  
K. Soe ◽  
H. Beard ◽  
D. Neumann ◽  
P. J. Trim ◽  
S. Duplock ◽  
...  
Keyword(s):  
Mucopolysaccharidosis Type ◽  
Disease Course ◽  
Early Disease ◽  
Type Iiia ◽  
Mps Iiia ◽  
Mucopolysaccharidosis Type Iiia

Design, baseline characteristics, and early findings of the MPS IIIA (mucopolysaccharidosis type IIIA) clinical observational study

2018 ◽  
Vol 123 (2) ◽  
pp. S113-S114
Author(s):  
Samantha Parker ◽  
Sophie Olivier ◽  
Philippe Drevot ◽  
Anupam Chakrapani ◽  
Benedicte Heron ◽  
...  
Keyword(s):  
Observational Study ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Baseline Characteristics ◽  
Mps Iiia ◽  
Mucopolysaccharidosis Type Iiia

Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA

2008 ◽  
Vol 93 (4) ◽  
pp. 411-418 ◽  
Author(s):  
Chantelle McIntyre ◽  
Ainslie Lauren Derrick Roberts ◽  
Enzo Ranieri ◽  
Peter Roy Clements ◽  
Sharon Byers ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia
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