Compositions and methods for treating or preventing hereditary angioedema

Described herein are compositions and methods for treating a subject having or at risk of developing hereditary angioedema. Using the compositions and methods of the disclosure a patient may be provided one or more agents that elevate the expression and/or activity levels of C1 -esterase inhibitor (...

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Bibliographische Detailangaben
Hauptverfasser: WOLFE, Jia L, MARKS-BLUTH, Jonathon Simeon, GASPAR, Bobby, SAGOO, Pervinder, LEMOINE, Adrien, PENNUCCI, Valentina
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung:Described herein are compositions and methods for treating a subject having or at risk of developing hereditary angioedema. Using the compositions and methods of the disclosure a patient may be provided one or more agents that elevate the expression and/or activity levels of C1 -esterase inhibitor (C1-INH). Exemplary agents that may be used in conjunction with the compositions and methods of the disclosure for this purpose include cells, such as pluripotent cells, that express C1- INH.