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

Abstract excerpt

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...

Topics

• Humans
• alpha 1-Antitrypsin Deficiency
• Genetic Therapy
• Gene Editing
• Animals
• alpha 1-Antitrypsin
• RNA Editing