From the World of BMT to ERT to GT - There is one shred of hope..... gene therapy trial conducted by a company known as Lysogene to treat patients with sanfilippo syndrome, who are missing a functional SGSH gene. The therapy works by injecting the patients brain with a human engineered virus (a virus programmed to not harm human tissue), carrying the SGSH gene. However, the virus also carries the SUMF-1 gene as well - the gene Izzy is deficient in! The reason they put the SUMF-1 gene in was to boost the activity of the SGSH enzyme, so theoretically if injected in an MSD patient it would replace the master key enzyme that would activate all the other sulfatase enzymes. for more detail read the post below and Help Izzy Lick Leukrodystrophy !
Posted on: Sun, 09 Feb 2014 19:33:45 +0000
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