Correction of mucopolysaccharidosis type IIIA somatic and central nervous system pathology by lentiviral‐mediated gene transfer
C McIntyre, S Byers, DS Anson - The journal of gene medicine, 2010 - Wiley Online Library
Background The hallmark of lysosomal storage disorders (LSDs) is microscopically
demonstrable lysosomal distension. In mucopolysaccharidosis type IIIA (MPS IIIA), this …
demonstrable lysosomal distension. In mucopolysaccharidosis type IIIA (MPS IIIA), this …
[HTML][HTML] Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice
FJ Duncan, BJ Naughton, K Zaraspe, DA Murrey… - Molecular Therapy, 2015 - cell.com
Mucopolysaccharidosis (MPS) IIIA is a neuropathic lysosomal storage disease caused by
deficiency in N-sulfoglucosamine sulfohydrolase (SGSH). Genome-wide gene expression …
deficiency in N-sulfoglucosamine sulfohydrolase (SGSH). Genome-wide gene expression …
Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral‐mediated gene delivery
C McIntyre, ALK Derrick‐Roberts… - The Journal of Gene …, 2014 - Wiley Online Library
Background Mucopolysaccharidoses (MPS) are inborn metabolic disorders caused by a
deficiency of glycosaminoglycan degrading enzymes. Although intravenous enzyme …
deficiency of glycosaminoglycan degrading enzymes. Although intravenous enzyme …
[HTML][HTML] Targeting the root cause of mucopolysaccharidosis IIIA with a new scAAV9 gene replacement vector
TA Bobo, PN Samowitz, MI Robinson, H Fu - Molecular Therapy-Methods & …, 2020 - cell.com
No treatment is available to address the unmet needs of mucopolysaccharidosis (MPS) IIIA
patients. Targeting the root cause, we developed a new self-complementary adeno …
patients. Targeting the root cause, we developed a new self-complementary adeno …
Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA
C McIntyre, ALD Roberts, E Ranieri… - Molecular genetics and …, 2008 - Elsevier
Mucopolysaccharidosis type IIIA (MPS IIIA) is a heritable glycosaminoglycan (GAG) storage
disorder which is characterised by lysosomal accumulation of heparan sulphate, secondary …
disorder which is characterised by lysosomal accumulation of heparan sulphate, secondary …
A preclinical study evaluating AAVrh10-based gene therapy for Sanfilippo syndrome
Mucopolysaccharidosis type IIIA (MPS IIIA) is predominantly a disorder of the central
nervous system, caused by a deficiency of sulfamidase (SGSH) with subsequent storage of …
nervous system, caused by a deficiency of sulfamidase (SGSH) with subsequent storage of …
[HTML][HTML] Overcoming limitations inherent in sulfamidase to improve mucopolysaccharidosis IIIA gene therapy
Y Chen, S Zheng, L Tecedor, BL Davidson - Molecular Therapy, 2018 - cell.com
Sulfamidase (SGSH) deficiency causes mucopolysaccharidosis type IIIA (MPS IIIA), a
lysosomal storage disease (LSD) that affects the CNS. In earlier work in LSD mice and dog …
lysosomal storage disease (LSD) that affects the CNS. In earlier work in LSD mice and dog …
[HTML][HTML] Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery
H Fu, MP Cataldi, TA Ware, K Zaraspe… - … Therapy Methods & …, 2016 - cell.com
The reversibility of neuropathic lysosomal storage diseases, including MPS IIIA, is a major
goal in therapeutic development, due to typically late diagnoses and a large population of …
goal in therapeutic development, due to typically late diagnoses and a large population of …
[HTML][HTML] Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery
H Fu, RJ Samulski, TJ McCown, YJ Picornell… - Molecular Therapy, 2002 - cell.com
Mucopolysaccharidosis (MPS) IIIB is characterized by mild somatic features and severe
neurological diseases leading to premature death. No definite treatment is available for MPS …
neurological diseases leading to premature death. No definite treatment is available for MPS …
[HTML][HTML] Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease
A Sergijenko, A Langford-Smith, AY Liao, CE Pickford… - Molecular Therapy, 2013 - cell.com
Mucopolysaccharidosis type IIIA (MPSIIIA) is a lysosomal storage disorder caused by
mutations in N-sulfoglucosamine sulfohydrolase (SGSH), resulting in heparan sulfate (HS) …
mutations in N-sulfoglucosamine sulfohydrolase (SGSH), resulting in heparan sulfate (HS) …