First Gene Therapy Applied Topically Delivers Results for DEB

Plus: Innovative Stargardt Gene Therapy Emerges

Author

Kris Tatiossian, PhD
April 23, 2023

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Welcome to all our new readers who came from Facebook after our video on CAR T cancer therapy went semi-viral! 🫶🏼 You’re just in time for 4 more awesome stories this week:

  • First Gene Therapy Applied Topically Delivers Results for DEB

  • Innovative Stargardt Gene Therapy Emerges READER REQUEST

  • Raziel Therapeutics Battles Dercum Disease READER REQUEST

  • CRISPR, Dorian Gray, and the Future of Youthfulness READER REQUEST

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🔴 First Gene Therapy Applied Topically Delivers Results

Dystrophic Epidermal Bullosa (DEB) is a terrible condition that causes the skin to be very fragile and blister easily.

1/ DEB is caused by mutations in the COL7A1 gene, which encodes for the collagen 7 protein. Collagen 7 is essential to attach our dermis and epidermis to each another. Therefore, mutations in this gene affect the dermis-epidermis cohesion leading to lifelong painful open wounds and blistering.

How does the topical gene therapy for DEB work? 👇🏼

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