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View article: Carnitine is a pharmacological allosteric chaperone of the human lysosomal <i>α</i>-glucosidase
Carnitine is a pharmacological allosteric chaperone of the human lysosomal <i>α</i>-glucosidase Open
Pompe disease is an inherited metabolic disorder due to the deficiency of the lysosomal acid α-glucosidase (GAA). The only approved treatment is enzyme replacement therapy with the recombinant enzyme (rhGAA). Further approaches like…
View article: Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease
Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease Open
View article: Carnitine is a pharmacological allosteric chaperone of the human lysosomal <i>α</i>-glucosidase
Carnitine is a pharmacological allosteric chaperone of the human lysosomal <i>α</i>-glucosidase Open
Pompe disease is an inherited metabolic disorder due to the deficiency of the lysosomal acid α-glucosidase (GAA). The only approved treatment is enzyme replacement therapy with the recombinant enzyme (rhGAA). Further approaches like…
View article: Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA
Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA Open
View article: 724. A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-Associated Virus Serotypes upon Cerebrospinal Fluid Administration in Pigs
724. A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-Associated Virus Serotypes upon Cerebrospinal Fluid Administration in Pigs Open
Gene transfer of recombinant adeno-associated virus (rAAVs) holds promises to treat neurological disorders. Studies on different recombinant vector serotypes and modes of delivery to the central nervous system (CNS) showed that the combina…
View article: Trop2 marks transient gastric fetal epithelium and adult regenerating cells after epithelial damage
Trop2 marks transient gastric fetal epithelium and adult regenerating cells after epithelial damage Open
Mouse fetal intestinal progenitors lining the epithelium prior to villogenesis grow as spheroids when cultured ex vivo and express the transmembrane glycoprotein Trop2 as a marker. In the present study, we report on the characterization of…
View article: A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs
A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs Open