Gene therapy for neuronopathic mucopolysaccharidoses: state of the art
MJ de Castro, M Del Toro, R Giugliani… - International Journal of …, 2021 - mdpi.com
The need for long-lasting and transformative therapies for mucopolysaccharidoses (MPS)
cannot be understated. Currently, many forms of MPS lack a specific treatment and in other …
cannot be understated. Currently, many forms of MPS lack a specific treatment and in other …
[HTML][HTML] Treatment of brain disease in the mucopolysaccharidoses
M Scarpa, PJ Orchard, A Schulz, PI Dickson… - Molecular genetics and …, 2017 - Elsevier
The mucopolysaccharidosis (MPS) disorders are a group of lysosomal storage diseases
caused by lysosomal enzyme deficits that lead to glycosaminoglycan accumulation, affecting …
caused by lysosomal enzyme deficits that lead to glycosaminoglycan accumulation, affecting …
Transplantation of Wild-Type Hematopoietic Stem and Progenitor Cells Improves Disease Phenotypes in Novel Mucopolysaccharidosis Iiic Mouse Model
RA Badell-Grau, K Pakravesh, KE Thai, F Son, R Chen… - papers.ssrn.com
Mucopolysaccharidosis type IIIC (MPSIIIC) is a severe neurodegenerative lysosomal
storage disease (LSD) caused by loss-of-function of the lysosomal transmembrane protein …
storage disease (LSD) caused by loss-of-function of the lysosomal transmembrane protein …
Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transfer
GRD Villani, A Follenzi, B Vanacore… - Biochemical …, 2002 - portlandpress.com
Mucopolysaccharidosis type IIIB (MPS IIIB; or Sanfilippo syndrome type B) is a lysosomal
disease, due to glycosaminoglycan storage caused by mutations on the α-N …
disease, due to glycosaminoglycan storage caused by mutations on the α-N …
610. High Level Expression of Human Iduronidase Throughout the Brain in a Murine Model of Mucopolysaccharidosis Type I After Non-Invasive AAV-Mediated Gene …
L Belur, Z Nan, M Buckvold, A Karlen, K Kitto… - Molecular Therapy, 2014 - cell.com
Mucopolysaccharidosis Type I (MPS I) is an inherited metabolic disease, caused by
deficiency of the lysosomal enzyme alpha-L-iduronidase (IDUA). Individuals with the most …
deficiency of the lysosomal enzyme alpha-L-iduronidase (IDUA). Individuals with the most …
Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse
TJ Sferra, K Backstrom, C Wang, R Rennard, M Miller… - Molecular Therapy, 2004 - cell.com
Mucopolysaccharidosis type VII is a lysosomal storage disease caused by deficiency of the
acid hydrolase β-glucuronidase. MPS VII mice develop progressive lysosomal accumulation …
acid hydrolase β-glucuronidase. MPS VII mice develop progressive lysosomal accumulation …
Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102, an intravenous AAV9-based gene therapy for Sanfilippo syndrome type A …
KM Flanigan, NJC Smith, ML Couce, KV Truxal… - 2020 - AAN Enterprises
Objective: To develop a gene therapy treatment for Mucopolysaccharidosis IIIA (MPS-IIIA)
based on intravenous administration of ABO-102. Background: MPS-IIIA is a lysosomal …
based on intravenous administration of ABO-102. Background: MPS-IIIA is a lysosomal …
[HTML][HTML] CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)
Mucopolysaccharidosis type II (MPSII) is an X-linked lysosomal storage disease
characterized by severe neurologic and somatic disease caused by deficiency of iduronate …
characterized by severe neurologic and somatic disease caused by deficiency of iduronate …
[引用][C] CAMPSIITE™ phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic …
P Harmatz, C Ficicioglu, R Giugliani, D Rajan… - Molecular Genetics and …, 2024 - Elsevier
[HTML][HTML] Disease modeling for Mucopolysaccharidosis type IIIB using patient derived induced pluripotent stem cells
Abstract Mucopolysaccharidosis type IIIB (MPS IIIB) is a lysosomal disease caused by
mutations in the NAGLU gene encoding α-N-acetylglucosaminidase (NAGLU) which …
mutations in the NAGLU gene encoding α-N-acetylglucosaminidase (NAGLU) which …