Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency
Horae Gene Therapy Center; Department of Pediatrics, Division of Pulmonary and Allergy
Genetics and Genomics | Molecular Biology | Therapeutics
This review seeks to give an overview of alpha-1 antitrypsin deficiency, including the different disease phenotypes that it encompasses. We then describe the different therapeutic endeavors that have been undertaken to address these different phenotypes. Lastly we discuss future potential therapeutics, such as genome editing, and how they may play a role in treating alpha-1 antitrypsin deficiency.
CRISPR/Cas9, Emphysema, Genome editing, Liver disease, SERPINA1
DOI of Published Version
Methods Mol Biol. 2017;1639:267-275. doi: 10.1007/978-1-4939-7163-3_27. Link to article on publisher's site
Methods in molecular biology (Clifton, N.J.)
Gruntman, Alisha and Flotte, Terence R., "Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency" (2017). Pediatric Publications and Presentations. 159.