134: The Science of Gene Editing & Treating Blindness

For the first time ever we’ve used CRISPR  to edit someone’s genes INSIDE THEIR BODY! So far we’ve been able to reverse some effects of rare genetic eye disease that results in blindness, and this is only the beginning!

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References & Further Reading

1. https://medlineplus.gov/genetics/condition/leber-congenital-amaurosis/
2. https://rarediseases.info.nih.gov/diseases/634/leber-congenital-amaurosis
3. https://luxturna.com

Gene Therapy

1. https://www.nature.com/articles/d41586-018-06195-6
2. https://www.nature.com/articles/s41587-020-0493-4
3. https://www.nature.com/articles/s41573-019-0012-9

The EDIT-101 Trial

1. https://clinicaltrials.gov/ct2/show/study/NCT03872479
2. https://www.nature.com/articles/s41591-018-0327-9
3. https://text.npr.org/2021/09/29/1040879179/vision-loss-crispr-treatment
4. https://www.nature.com/articles/d41586-020-00655-8https://www.nature.com/articles/d41586-020-00655-8
5. https://jamanetwork.com/journals/jama/fullarticle/2764428https://jamanetwork.com/journals/jama/fullarticle/2764428