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The underlying cause of MPS IIIA is a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate throughout the body resulting in rapid decline associated with the disorder.
But the Manchester team developed a way of over-producing the SGSH enzyme specifically in bone marrow white blood cells.
The lentiviral vector delivers the SGSH gene to bone marrow cells.
Bigger said to increase SGSH enzyme from bone marrow transplants, and to target it to the cells that traffic into die brain, the researchers developed a stem cell gene therapy which overproduces the SGSH enzyme specifically in bone marrow white blood cells.
The protests led by the leadership of young doctors were held near Pakistan Institute of Medical Sciences (PIMS) and on Fazl-e-Haq road near Federal Government Services Hospital (SGSH).
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- SGS Portable Integrated Data Extraction and Reduction
- SGS-Thompson Microelectronics