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Gain Gene Replacement Therapy Equipment & Supplies
2 equipment items found
by:Gain Therapeutics, Inc. based inBethesda, MARYLAND (USA)
Morquio B, also known as Mucopolysaccharidosis type IV (MPS IV), is a progressive disease mostly impacting the skeleton, caused by mutations in GLB1, the gene that encodes the beta-galactosidase (GLB) enzyme. These mutations result in the misfolding and subsequent dysfunction of GLB, which leads to the toxic substrate accumulation of keratan sulfate in organs and tissues. Very ...
by:Gain Therapeutics, Inc. based inBethesda, MARYLAND (USA)
GM1 Gangliosidosis is a hereditary, progressive disease mostly impacting neurons in the brain and spinal cord, caused by mutations in GLB1, the gene that encodes the beta-galactosidase (GLB) enzyme. These mutations result in the misfolding and subsequent dysfunction of GLB, which leads to the toxic substrate accumulation of GM1 ganglioside in organs and tissues. Very limited and ...