Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema

Two patients with severe bradykinin-mediated angioedema received weekly subcutaneous injections of antisense oligonucleotides targeting prekallikrein messenger RNA. Subsequent to drug administration, they had reduced attack rates.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2020-09, Vol.383 (13), p.1242-1247
Hauptverfasser:	Cohn, Danny M, Viney, Nicholas J, Fijen, Lauré M, Schneider, Eugene, Alexander, Veronica J, Xia, Shuting, Kaeser, Gwendolyn E, Nanavati, Charvi, Baker, Brenda F, Geary, Richard S, Levi, Marcel, Meijers, Joost C.M, Stroes, Erik S.G
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Angioedema Angioedemas, Hereditary - drug therapy Angioedemas, Hereditary - metabolism Antisense oligonucleotides Antisense therapy Bradykinin Bradykinin - metabolism Catheters Compassionate Use Trials Edema Female Hepatocytes Humans Injections, Subcutaneous Mucosa Oligonucleotides, Antisense - administration & dosage Oligonucleotides, Antisense - therapeutic use Patients Pilot Projects Plasma Prekallikrein - antagonists & inhibitors Prekallikrein - metabolism
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Beschreibung
Zusammenfassung:	Two patients with severe bradykinin-mediated angioedema received weekly subcutaneous injections of antisense oligonucleotides targeting prekallikrein messenger RNA. Subsequent to drug administration, they had reduced attack rates.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa1915035