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Gene Therapy Gel Offers New Hope Against Rare Blistering Disease

December 15, 2022

Via: Drugs.com

An experimental gene therapy that’s applied as a skin gel appears to heal wounds caused by a rare and severe genetic skin disease.

Experts called the findings “remarkable,” and said they bring hope of a better quality of life to children and young adults living with the condition, called dystrophic epidermolysis bullosa (DEB).

The disease affects about 3 out of every 1 million people. It’s caused by a flawed gene that renders the body unable to produce a particular collagen — a “glue” between the skin layers that is essential to its strength and integrity.

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