Enzymatic nanoreactors were obtained by galactose-1-phosphate uridylyl-transferase (GALT) encapsulation into plant virus capsids by a molecular self-assembly strategy. The aim of this work was to produce virus-like nanoparticles containing GALT for an enzyme-replacement therapy for classic galactosemia. The encapsulation efficiency and the catalytic constants of bio-nanoreactors were determined by using different GALT and virus coat protein ratios. The substrate affinity of nanoreactors was slightly lower than that of the free enzyme; the activity rate was 16 % of the GALT free enzyme. The enzymatic nanoreactors without functionalization were internalized into different cell lines including fibroblast and kidney cells, but especially into hepatocytes. The enzymatic nanoreactors are an innovative enzyme preparation with potential use for the treatment of classic galactosemia.
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http://dx.doi.org/10.1002/cmdc.202000999 | DOI Listing |
J Clin Pharmacol
November 2024
Applied Therapeutics Inc., New York, NY, USA.
Biomed Pharmacother
December 2024
Centro Andaluz de Biología del Desarrollo (CABD) - Universidad Pablo de Olavide (UPO), Departamento de Biología Molecular e Ingeniería Bioquímica, UPO/CSIC/JA, Sevilla 41013, Spain. Electronic address:
Type III galactosemia is characterized by the inability to metabolize galactose due to deficiency of the UDP-galactose-4-epimerase (GALE) gene, which catalyzes the interconversion of UDP-Galactose and UDP-Glucose. Additionally, GALE interconverts UDP-N-Acetylgalactosamine and UDP-N-Acetylglucosamine. These four sugars are needed for glycosylation of biomolecules.
View Article and Find Full Text PDFClin Transl Med
October 2024
Division of Pediatric and Adolescent Gynecology, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, Maryland, USA.
BMC Pediatr
September 2024
Diagnosis and Treatment Unit of Congenital Metabolic Diseases (UDyTEMC), Clinical University Hospital of Santiago de Compostela. National Reference Center for Metabolic Diseases (C.S.U.R.), Santiago de Compostela, Spain.
Orphanet J Rare Dis
September 2024
Univ. Savoie Mont Blanc, Univ. Grenoble Alpes, LIP/PC2S, Grenoble, 38000, France.
Background: Classic galactosemia is a rare inherited metabolic disease with long-term complications, particularly in the psychosocial domain. Patients report a lower quality of social life, difficulties in interactions and social relationships, and a lower mental health. We hypothesised that social cognition deficits could partially explain this psychological symptomatology.
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