Evaluation of HexM and its high-level mannose 6-phosphorylated form, PhosHexM, as treatments of models of GM2 gangliosidoses

This publication has 35 references indexed in Scilit:

• Evaluating the state of the science for adeno-associated virus integration: An integrated perspective
  Molecular Therapy, 2022
• Increased phosphorylation of HexM improves lysosomal uptake and potential for managing GM2 gangliosidoses
  BBA Advances, 2022
• Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice
  Molecular Therapy, 2020
• A low-cost and customizable alternative for commercial implantable cannula for intracerebral administration in mice
  HardwareX, 2020
• Disease‐causing missense mutations within the N‐terminal transmembrane domain of GlcNAc‐1‐phosphotransferase impair endoplasmic reticulum translocation or Golgi retention
  Human Mutation, 2020
• Murine Sialidase Neu3 facilitates GM2 degradation and bypass in mouse model of Tay-Sachs disease
  Experimental Neurology, 2018
• Engineering of GlcNAc-1-Phosphotransferase for Production of Highly Phosphorylated Lysosomal Enzymes for Enzyme Replacement Therapy
  Molecular Therapy - Methods & Clinical Development, 2017
• Highly phosphomannosylated enzyme replacement therapy for GM2 gangliosidosis
  Annals of Neurology, 2010
• Quantitative correlation between the residual activity of β-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease
  Human Genetics, 1992
• Natural History and Inherited Disorders of a Lysosomal Enzyme, β-Hexosaminidase
  Journal of Biological Chemistry, 1989