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In: Conn's Current Therapy 2020

Mesenchymal stem cells

However, in a large study at a poison center, only 5% of patients ultimately required intravenous therapy because they could not tolerate the oral agent

The expression of the remaining 18 genes, including ORC4 , BCL11B , and PAG1 , which were upregulated in the patients not treated with L-serine, was reduced following L-serine treatment

It is thought that delivery and expression of the FXN gene will complement the loss of FXN expression from the mutant alleles and potentially reverse FRDA phenotypes