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Mucopolysaccharidosis type IIIB (MPS IIIB) is a lysosomal disease, an inborn error of metabolism.

Mucopolysaccharidosis type IIIB (MPS IIIB) is a lysosomal disease, an inborn error of metabolism.

Dhaka June 5, 2015. Enzyme-IGFII fusion protein next therapeutic agent for Mucopolysaccharidosis type IIIB Mucopolysaccharidosis type IIIB (MPS IIIB) is a lysosomal disease, an inborn error of metabolism. It mainly affects the brain and spinal cord. Affected individuals can face some neurological symptoms, including dementia, aggressive behavior, hyperactivity, loss of vision, and can’t even sleep for more than a few hours at a time. Mika Aoyagi-Scharber , Patricia I. Dickson and Elizabeth F. Neufeld led a research jointly (doi: 10.1073/pnas.1416660111) where they worked on MPS IIIB in rat model and suggested a treatment for this diseases First of all, they made a fusion protein (“enzyme”) of recombinant alpha-N-acetylglucosaminidase (NAGLU) fused with insulin-like growth factor II(IGFII). They also prepared a artificial cerebrospinal fluid(CSF) for using as a vehicle. They injected the NAGLU-IGFII into the left ventricle of the brain. The protein was uptaken by the cells and started performing. As a result the heparan sulfate and specially, the disease-specific heparan sulfate was reduced remarkably. It also made some effect on the others disease-associated biomarkers like beta-Hexosaminidase, LAMP1, beta-amyloid, glypican 5, hyperphosphorylated tau (P-thr181) by decreasing them. In future, if we get this fusion protein into a useable form for human, then we will hope to control this MPS IIIB disease.

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