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5 Companies unraveling the potential of Gene Based Therapies for the treatment of Sanfilippo syndrome (Mucopolysaccharidosis type III (MPS III)

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is caused by the mutations in the glucosamine (N-acetyl)-6-sulfatase (GNS), heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT), N-acetyl-alpha-glucosaminidase (NAGLU) and N-sulfoglucosamine sulfohydrolase (SGSH) genes, which are responsible for the synthesis of large sugar molecules called glycosaminoglycans (GAGs). The dysfunction...

Gene Therapy offers Cure for Bubble Boy Syndrome

Severe combined immunodeficiency (SCID) or “Bubble Boy Syndrome” is a rare genetic disorder caused by gene mutations that inhibit the functioning of immune cells. Children afflicted with this condition are healthy in appearance but are susceptible to severe infections, even a common cold can prove...

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