Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency
Gruntman, Alisha M Flotte, Terence R.
Gruntman, Alisha M
Flotte, Terence R.
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UMass Chan Affiliations
Document Type
Book Chapter
Publication Date
2017-07-28
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Abstract
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.
Source
Methods Mol Biol. 2017;1639:267-275. doi: 10.1007/978-1-4939-7163-3_27. Link to article on publisher's site
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DOI
10.1007/978-1-4939-7163-3_27
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PubMed ID
28752467