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Current gene therapy research aims to target, for example, upstream targets of the GSH system or molecules associated with it (such as Nrf2, GPX, SLC7A11), genes aberrantly expressed in myocardial injury, and noncoding RNAs regulating mRNA transcription [76, 202, 250,251,252,253]

There may also be a lack of interest by pharmaceutical companies in pursuing drug development against NTDs due to a complicated profit motive [7]

As mentioned above, mutation of the latter residues leads to a pronounced lowering of the MGST1 activity and mutation of the former leads to an activity decrease as well as a 67 fold increase in K m for GSH (Table 1)

AAV-AADC phase-I clinical trials demonstrated safety and a significant improvement of both motor and non-motor symptoms as assessed by the Unified Parkinson Disease Rating Scale (UPDRS), a decrease in OFF-time duration without an increased effect of ON-time dyskinesias, as well as an increase in the uptake of the AADC tracer at PET, which was used as a measure of gene expression (Christine et al

4 weeks 28 syringes 1 per day