5 Articles found
Sigilon Therapeutics, Inc. Articles
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Development of a Novel Encapsulated Non-Viral Cell- Based, BBB-Penetrant Therapy for MPS I
Introduction MPS I is caused by a deficiency of the lysosomal enzyme a-L-iduronidase (IDUA) leading to GAG accumulation in multiple tissues and organs This accumulation results in a complex array of progressive, multi-systemic pathologies, ...
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Development of a Novel Encapsulated Non-Viral Cell- Based Therapy for MPS VI
Introduction Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) is caused by a deficiency of the lysosomal enzyme arylsulfatase B (ARSB) ARSB deficiency results in incomplete or blocked degradation of glucosaminoglycans (GAGs), ...
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Development of a Novel Encapsulated Non-Viral Cell-Based, BBB-Penetrant Therapy for MPS I
MPS I is caused by deficiency of the lysosomal enzyme a-L-iduronidase (IDUA) leading to GAG accumulation in multiple tissues and organs This accumulation results in a complex array of progressive, multi-systemic pathologies, including CNS ...
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SIG-005: Novel Encapsulated Non-Viral Cell-Based Therapy for MPS-1
HYPOTHESIS: sustained therapeutic effect could be achieved by administration of IDUA-secreting allogeneic human cells shielded within spheres designed to avoid immune rejection and pericapsular fibrotic overgrowth (PFO)
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Development of a novel encapsulated non-viral cell-based therapy for MPS-6
Introduction Mucopolysaccharidosis type VI (MPS-6, Maroteaux-Lamy syndrome) is caused by a deficiency of the lysosomal enzyme arylsulfatase B (ARSB) ARSB deficiency results in incomplete or blocked degradation of glycosaminoglycans (GAGs), which ...