Through ‘Prime Editing’, over 90% of inherited diseases can be targeted and corrected.
Targeting inherited retinal diseases in initial phases. ~80 different gene mutations cause Retinitis Pigmentosa and eventual blindness in patients. What if we created cell banks and expand drug outcomes?
After prime edits confirmed in iPSCs, downstream differentiation into RPE cell monolayers or other neural tissue to study disease progression. Displayed is a RPE monolayer after prime edits.
Creating different organoids and neural tissues will expound our knowledge deep into the cosmos. Displayed is a 150 day retinal organoid after prime edits.
Creating diseased cell lines and organoids will allow researchers to develop innovative therapies. Precision medicine and deep sequencing analysis through long read isoform capture is of interest and progressing forward through NSF grants.
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