Article
Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema.
Allergy - 1 Jun 2019
Qiu Ting, Chiuchiolo Maria J, Whaley Adele S, Russo Anthony R, Sondhi Dolan, Kaminsky Stephen M, Crystal Ronald G, Pagovich Odelya E
Abstract excerpt
BACKGROUND: Hereditary angioedema (HAE) is a life-threatening, autosomal dominant disorder characterized by unpredictable, episodic swelling of the face, upper airway, oropharynx, extremities, genitalia, and gastrointestinal tract. Almost all cases of HAE are caused by mutations in the SERPING1 gene resulting in a deficiency in functional plasma C1 esterase inhibitor (C1EI), a serine protease inhibitor that...
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