- What modern technologies, from gene therapy to PCR testing, depend on the decoded genetic alphabet?
Gene Therapy
Treating disease by introducing, altering, or removing genetic material.
- What kinds of diseases are now treated using RNAi-based therapies?
- What diseases have CRISPR-based therapies already reached clinical trials for?
- How was a functional copy of the OTOF gene delivered directly to the hair cells of the cochlea?
- How much did hearing thresholds improve in trial participants after the gene therapy injection?
- How does the RPE65 gene therapy for inherited retinal dystrophy work, and what does the corrected virus actually deliver to the retina?