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• W2885378797 abstract "Abstract 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 SERPING 1 gene resulting in a deficiency in functional plasma C1 esterase inhibitor (C1 EI ), a serine protease inhibitor that normally inhibits proteases in the contact, complement, and fibrinolytic systems. Current treatment of HAE includes long‐term prophylaxis with attenuated androgens or human plasma‐derived C1 EI and management of acute attacks with human plasma‐derived or recombinant C1 EI , bradykinin, and kallikrein inhibitors, each of which requires repeated administration. As an approach to effectively treat HAE with a single treatment, we hypothesized that a one‐time intravenous administration of an adeno‐associated virus ( AAV ) gene transfer vector expressing the genetic sequence of the normal human C1 esterase inhibitor ( AAV rh.10hC1 EI ) would provide sustained circulating C1 EI levels sufficient to prevent angioedema episodes. Methods To study the efficacy of AAV rh.10hC1 EI , we used CRISPR /Cas9 technology to create a heterozygote C1 EI ‐deficient mouse model (S63±) that shares characteristics associated with HAE in humans including decreased plasma C1 EI and C4 levels. Phenotypically, these mice have increased vascular permeability of skin and internal organs. Results Systemic administration of AAV rh.10hC1 EI to the S63± mice resulted in sustained human C1 EI activity levels above the predicted therapeutic levels and correction of the vascular leak in skin and internal organs. Conclusion A single treatment with AAV rh.10hC1 EI has the potential to provide long‐term protection from angioedema attacks in affected individuals." @default.
• W2885378797 created "2018-08-22" @default.
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• W2885378797 date "2019-03-19" @default.
• W2885378797 modified "2023-10-17" @default.
• W2885378797 title "Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema" @default.
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• W2885378797 doi "https://doi.org/10.1111/all.13582" @default.
• W2885378797 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/6752709" @default.
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• W2885378797 hasPublicationYear "2019" @default.
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