دورية أكاديمية
Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema.
العنوان: | Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema. |
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المؤلفون: | Riedl MA; Division of Allergy and Immunology, University of California, San Diego, La Jolla, Calif. Electronic address: mriedl@health.ucsd.edu., Bordone L; Ionis Pharmaceuticals, Carlsbad, Calif., Revenko A; Ionis Pharmaceuticals, Carlsbad, Calif., Newman KB; Ionis Pharmaceuticals, Carlsbad, Calif., Cohn DM; Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands. |
المصدر: | The journal of allergy and clinical immunology. In practice [J Allergy Clin Immunol Pract] 2024 Apr; Vol. 12 (4), pp. 911-918. Date of Electronic Publication: 2023 Dec 22. |
نوع المنشور: | Journal Article |
اللغة: | English |
بيانات الدورية: | Publisher: Elsevier Inc Country of Publication: United States NLM ID: 101597220 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2213-2201 (Electronic) NLM ISO Abbreviation: J Allergy Clin Immunol Pract Subsets: MEDLINE |
أسماء مطبوعة: | Original Publication: New York, NY : Elsevier Inc., [2013]- |
مواضيع طبية MeSH: | Angioedemas, Hereditary*/genetics , Angioedemas, Hereditary*/prevention & control, Humans ; Bradykinin/therapeutic use ; Bradykinin/metabolism ; Complement C1 Inhibitor Protein/therapeutic use ; Liver/metabolism ; Prekallikrein |
مستخلص: | Hereditary angioedema (HAE) is typically caused by a deficiency of the protease inhibitor C1 inhibitor (C1INH). The absence of C1INH activity on plasma kallikrein and factor XIIa leads to overproduction of the vasoactive peptide bradykinin, with resulting angioedema. As the primary site of C1INH and prekallikrein production, the liver is recognized as an important therapeutic target in HAE, leading to the development of hepatic-focused treatment strategies such as GalNAc-conjugated antisense technology and gene modification. This report reviews currently available data on hepatic-focused interventions for HAE that have advanced into human trials. Donidalorsen is an investigational GalNAc (Copyright © 2024 American Academy of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved.) |
فهرسة مساهمة: | Keywords: ASO; Antisense oligonucleotide; CRISPR/Cas systems; Gene therapy; Hereditary angioedema; Liver; siRNA |
المشرفين على المادة: | S8TIM42R2W (Bradykinin) 0 (Complement C1 Inhibitor Protein) 9055-02-1 (Prekallikrein) |
تواريخ الأحداث: | Date Created: 20231224 Date Completed: 20240409 Latest Revision: 20240426 |
رمز التحديث: | 20240427 |
DOI: | 10.1016/j.jaip.2023.12.025 |
PMID: | 38142864 |
قاعدة البيانات: | MEDLINE |
تدمد: | 2213-2201 |
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DOI: | 10.1016/j.jaip.2023.12.025 |