An LNP-CRISPR gene editing drug demonstrates efficacy and safety in patients with hereditary angioedema following in vivo administration.

Pardi, Norbert; Ivics, Zoltán

An LNP-CRISPR gene editing drug demonstrates efficacy and safety in patients with hereditary angioedema following in vivo administration.

Pardi, Norbert; Ivics, Zoltán.

Afiliación

Pardi N; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pa.
Ivics Z; Institute of Clinical Immunology, University of Leipzig, Leipzig, Germany; Fraunhofer Institute for Cell Therapy and Immunology, Leipzig, Germany. Electronic address: zoltan.ivics@izi.fraunhofer.de.

J Allergy Clin Immunol ; 154(2): 272-274, 2024 Aug.

Article en En | MEDLINE | ID: mdl-38925443

Asunto(s)

Angioedemas Hereditarios; Edición Génica; Humanos; Angioedemas Hereditarios/genética; Angioedemas Hereditarios/tratamiento farmacológico; Femenino; Masculino; Adulto; Persona de Mediana Edad; Sistemas CRISPR-Cas

Palabras clave

CRISPR/Cas9; Gene editing; gene knockout; hereditary angioedema; in vivo gene delivery; kallikrein; lipid nanoparticle

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Angioedemas Hereditarios / Edición Génica Límite: Adult / Female / Humans / Male / Middle aged Idioma: En Revista: J Allergy Clin Immunol Año: 2024 Tipo del documento: Article

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