Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema.
ASO
CRISPR-Cas systems
antisense oligonucleotide
gene therapy
hereditary angioedema
liver
siRNA
Journal
The journal of allergy and clinical immunology. In practice
ISSN: 2213-2201
Titre abrégé: J Allergy Clin Immunol Pract
Pays: United States
ID NLM: 101597220
Informations de publication
Date de publication:
22 Dec 2023
22 Dec 2023
Historique:
received:
27
07
2023
revised:
09
11
2023
accepted:
08
12
2023
medline:
25
12
2023
pubmed:
25
12
2023
entrez:
24
12
2023
Statut:
aheadofprint
Résumé
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 being recognized as an important therapeutic target in HAE, leading to development of hepatic-focused treatment strategies such as GalNAc-conjugated antisense technology and gene modification. This paper reviews currently available data on hepatic-focused interventions for HAE that have advanced into human trials. Donidalorsen is an investigational GalNAc
Identifiants
pubmed: 38142864
pii: S2213-2198(23)01388-0
doi: 10.1016/j.jaip.2023.12.025
pii:
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
Copyright © 2023. Published by Elsevier Inc.