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Fig. 5
Schematic of therapeutic development pipeline described and followed in this study.
Using this strategy we were able to develop, test, and validate enough product to treat 500 patients (at a maximum clinical dose of 1 × 1011v.g.) with MAK associated RP for less than $500,000 USD (i.e., ~$1000 USD/patient). v.g. = viral genomes. QC = quality control. Example QC analysis performed at each step is provided. For a complete list of QC analysis performed see Supplemental Table 3.
Acknowledgments
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