Preclinical enzyme replacement therapy with a recombinant -galactosidase-lectin fusion for cns delivery and treatment of gm1-gangliosidosis

HIGHLIGHTS

• who: Jason Andrew Weesner et al. from the Department of Genetics, StJude Children's Research Hospital, Memphis, TN, USA Department of Anatomy and Physiology, University of Tennessee Health Science Center have published the research: Preclinical Enzyme Replacement Therapy with a Recombinant -Galactosidase-Lectin Fusion for CNS Delivery and Treatment of GM1-Gangliosidosis, in the Journal: Cells 2022, 11, 2579. of /2022/
• what: The authors show that long-term bi-weekly systemic injection of mβ-Gal:RTB in the β-Gal-/- mouse model resulted in widespread internalization of the enzyme by cells of visceral organs with consequent . . .
  

   

  

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