Biochemical features of primary cells from a pediatric patient with a gain-of-function ODC1 genetic mutation

In conclusion, our patient and potentially other patients that carry a similar ODC1 gain-of-function mutation might benefit from treatment with DFMO, a drug with a good safety profile, to suppress the exceptionally high ODC activity and putrescine levels in the body.

Source: Biochemical Journal - July 23, 2019 Category: Biochemistry Authors: Schultz, C. R., Bupp, C. P., Rajasekaran, S., Bachmann, A. S. Tags: Research Articles Source Type: research