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CRISPR-Based Therapy for Hereditary Angioedema

by Shah, Mrinal Y. , Magerl, Markus , Golden, Adele , Launay, David , Abdelhady, Ahmed M. , Lebwohl, David , Petersen, Remy S. , Lindsay, Karen , Xu, Yuanxin , Longhurst, Hilary J. , Cohn, Danny M. , Katelaris, Constance H. , Aygören-Pürsün, Emel , Gurugama, Padmalal , Maag, David , Butler, James S. , Bouillet, Laurence

in Administration, Intravenous / Adolescent / Adult / Aged / Airway management / Angioedema / Angioedemas, Hereditary - blood / Angioedemas, Hereditary - diagnosis / Angioedemas, Hereditary - genetics / Angioedemas, Hereditary - therapy / CRISPR / CRISPR-Associated Protein 9 - genetics / CRISPR-Cas Systems - genetics / Double-Blind Method / Female / Gene Editing / Gene Editing - methods / Genetic disorders / Genetic Therapy - adverse effects / Genetic Therapy - methods / Genetics / Genetics General / Hematology / Humans / Immunity / Kallikreins / Kallikreins - blood / Kallikreins - genetics / Life Sciences / Male / Middle Aged / Oncology / Oncology General / Patients / Pharmacodynamics / Pharmacokinetics / Placebos / Plasma / Plasma kallikrein / Proteins / Quality of life / Rhinopharyngitis / RNA, Guide, CRISPR-Cas Systems - administration & dosage / RNA, Guide, CRISPR-Cas Systems - adverse effects / RNA, Guide, CRISPR-Cas Systems - genetics / Statistical analysis / Treatment Outcome / Young Adult

2025

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2025

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2025

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Journal Article

CRISPR-Based Therapy for Hereditary Angioedema

Shah, Mrinal Y.,

Magerl, Markus,

Golden, Adele,

Launay, David,

Abdelhady, Ahmed M.,

Lebwohl, David,

Petersen, Remy S.,

Lindsay, Karen,

Xu, Yuanxin,

Longhurst, Hilary J.,

Cohn, Danny M.,

Katelaris, Constance H.,

Aygören-Pürsün, Emel,

Gurugama, Padmalal,

Maag, David,

Butler, James S.,

Bouillet, Laurence

2025

Overview

In this randomized, controlled trial, the number of angioedema attacks per month was approximately 75% lower among adults with hereditary angioedema who received a CRISPR-Cas9–based therapy than among those who received placebo.

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Publisher

Massachusetts Medical Society

Subject

Administration, Intravenous

/ Adolescent

/ Adult

/ Aged

/ Airway management

/ Angioedema

/ Angioedemas, Hereditary - blood