Advances in Precision Editing Therapies for Alpha-1 Antitrypsin Deficiency.

Jenny Gao; Erik Sontheimer; Terence R Flotte; Wen Xue

doi:10.1177/10430342261417356

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Advances in Precision Editing Therapies for Alpha-1 Antitrypsin Deficiency.

Jenny Gao·Erik Sontheimer·Terence R Flotte·Wen Xue

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Abstract

Genome and RNA editing modalities have revolutionized precision gene therapy, offering a safer alternative to traditional gene replacement approaches. Alpha-1 antitrypsin deficiency (AATD) is a compelling model for precision medicine because the disease mechanism is well defined-mutations in a single gene are responsible for both liver and lung pathology. In this review, we summarize the current preclinical and clinical efforts for AATD, with an emphasis on genome and RNA editing strategies.

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Humans alpha 1-Antitrypsin Deficiency Genetic Therapy Gene Editing Animals alpha 1-Antitrypsin RNA Editing Precision Medicine Mutation RNA editing alpha-1 antitrypsin base editing gene editing prime editing Journal Article Review

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Jenny Gao, Erik Sontheimer, Terence R Flotte, Wen Xue. 2026-01-26. Advances in Precision Editing Therapies for Alpha-1 Antitrypsin Deficiency.. https://doi.org/10.1177/10430342261417356

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