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Gene Transfer into Human Hematopoietic Stem Cells
Published in Richard K. Burt, Alberto M. Marmont, Stem Cell Therapy for Autoimmune Disease, 2019
Serguei Kisselev, Tatiana Seregina, Richard K. Burt, Charles J. Link
Fabry disease is a human lysosomal storage disorder due to an inborn defect of the alpha-galactosidase A gene. Preliminary experiments demonstrated that cord blood or bone marrow CD34+ cells as well as long-term culture derived progenitor cells could be successfully transduced by retroviral vectors carrying the alpha-gal A transgene.117 The efficiency of cord blood CD34+ transduction in this pre-clinical study ranged from 34% to 82%. Furthermore, in vitro experiments established that alpha-gal A transduced cells could secrete alpha-galactosidase A and complement the genetic defect of non-transduced cells derived from patients with Fabry disease.
Biocatalytic Nanoreactors for Medical Purposes
Published in Peter Grunwald, Pharmaceutical Biocatalysis, 2019
Oscar González-Davis, Chauhan Kanchan, Rafael Vazquez-Duhalt
Fabry disease is a disorder linked to glycosphingolipid metabolism caused by α-galactosidase A (α-Gal) deficiency. The successful use of ERT for Fabry disease has been reported (Alfadhel and Sirrs, 2011). Recombinant agalsidase alfa and agalsidase beta have been compared for Fabry disease treatment (Arends et al., 2018). The authors found no difference between patients treated with either agalsidase, and a more pronounced reduction of storage materials was found with agalsidase beta.
Production, purification, characterization, and applications of α-galactosidase from Bacillus flexus JS27 isolated from Manikaran hot springs
Published in Preparative Biochemistry & Biotechnology, 2023
Sonu Bhatia, Navneet Batra, Jagtar Singh
α-Galactosidase is an exceptionally important industrial enzyme used in food/feed, guar gum, and biomass processing. In the food processing industry, the enzyme is employed in the removal of raffinose family oligosaccharides (RFOs) as they are not hydrolyzed by humans and monogastric animals due to lack of pancreatic α-galactosidase in their intestine.[4,5] Functional food ingredients galactooligosaccharides (GOS) are produced by α-galactosidase through transgalactosylation. GOS possesses prebiotic properties which can encourage the growth of probiotic bacteria, preventing the growth of enteric pathogens thus improving the host’s overall health.[6] The enzyme has been studied extensively for its role in Fabry disease and its treatment using enzyme replacement therapy (ERT). Other applications include its use as a digestive aid, conversion of blood groups, and prevention of xenograft rejection.[3]