In this episode, Dr. Mark Pennesi provides an overview of inherited retinal dystrophies and how CRISPR is being used to edit DNA in the eyes of patients. Dr. Pennesi further shares recent research findings from a clinical trial using CRISPR to treat patients with Leber Congenital Amaurosis.
For more information on the guest, check out our website: broadeye.org/pennesi/
00:00 Intro
0:29 CRISPR Technology: What it is and how it works
4:29 Diseases treated with Gene Editing
8:49 Leber congenital amaurosis and Gene Editing
13:18 The BRILLIANCE Trial
16:20 The First Gene Editing in Vivo
18:48 Gene Editing and other forms of inherited retinal disease
22:09 The next steps
23:19 Conclusion
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