Gene therapy for AGU mice

Here are few articles that I found referring to gene therapy trials in AGU mice. Looks like all were pretty successful.

• Enomaa et al

"We have successfully performed enzyme replacement and gene therapy (with retrovirus-mediated transfer of the AGA gene) both in AGU patients primary fibroblasts and in neural cells of murine origin. Cross-correction between treated and untreated cells could also be achieved, suggesting that only a relatively small number of treated cells would be sufficient to correct the deficient enzyme activity in vivo."

• Harkee et al

AGA, like lysosomal enzymes in general, are good targets for gene therapy since they move from cell to cell using the mannose-6-phosphate receptor. Consequently, only a minority of target cells need to be corrected. Here, we wanted to determine which cell type, neurons or glia would better produce AGA to be transported to adjacent cells for use in possible treatment strategies. Adenoviruses containing tissue-specific glial fibrillary acidic protein (GFAP) promoter and neuron-specific enolase (NSE) promoter were generated to target expression of AGA in Aga-deficient mouse primary glial and neuronal cell cultures. AGA promoter was shown to be a very powerful glia promoter producing 32 times higher specific AGA activity in glia than in neurons. GFAP and NSE promoters also produced a clear overexpression of AGA in glia and neurons, respectively. Interestingly, both the NSE and GFAP promoters were not cell-specific in our system. The amount of exocytosed AGA was significantly higher in glial cells than neurons and glial cells were also found to have a greater capacity to endocytose AGA.

Degradation and Turnover of Glycans

For me, not having a biological background, sites that give explanations of the complex and logical biological information for free are always a pot of gold.

Here is a very interesting one that I read today: Degradation and Turnover of Glycans.

It gave me the explanation of glycoprotein degradation sequence, and also have other interesting information.