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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Bibliographic Details
Main Authors SAGOO, PERVINDER, MARKS-BLUTH, JONATHON SIMEON, WOLFE, JIA, GASPAR, BOBBY, PENNUCCI, VALENTINA, LEMOINE, ADRIEN
Format Patent
LanguageEnglish
French
Published 17.02.2022
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Summary: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. La présente invention concerne des compositions et des méthodes pour traiter un sujet présentant ou risquant de développer un angio-dème héréditaire. Les compositions et les méthodes selon l'invention permettent d'administrer à un patient un ou plusieurs agents destinés à augmenter les niveaux d'expression et/ou d'activité de l'inhibiteur de la C1 estérase (C1-INH). Des exemples d'agents qui peuvent être utilisés conjointement avec les compositions et les méthodes selon l'invention à cet effet comprennent des cellules, telles que des cellules pluripotentes, exprimant le C1-INH.
Bibliography:Application Number: CA20213191729