Episode 147: CEP290 mRNA intravitreal therapy for LCA

This week’s episode focuses on a recent publication (https://www.nature.com/articles/s41591-018-0295-0) in Nature Medicine concerning the successful use of an intravitreal oligonucleotide for a form of Leber Congential Amaurosis (LCA). First, Dr. Allen Ho, one of the authors of the paper, rejoins the program to discuss the project and its implications. Then Dr. Matthew Weed returns to offer his perspective as a ophthalmic genetics specialist on this trial compared to other modalities such as Luxturna and what it means for the future. Dr Weed and Dr. Sridhar have no relevant financial disclosures. Dr. Ho has receieved grant funding from Pro QR.

Click here to view our “Lessons from our Pupils” Blog Post related to this episode!