Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency

Authors
Gruntman, Alisha
Flotte, Terence R.
UMass Chan Affiliations
Department of Pediatrics, Division of Pulmonary and Allergy
Horae Gene Therapy Center
Document Type
Book Chapter
Publication Date
2017-07-28
Keywords
CRISPR/Cas9
Emphysema
Genome editing
Liver disease
SERPINA1
Genetics and Genomics
Molecular Biology
Therapeutics

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Link to Full Text
https://doi.org/10.1007/978-1-4939-7163-3_27
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
DOI
10.1007/978-1-4939-7163-3_27
Permanent Link to this Item
http://hdl.handle.net/20.500.14038/43589
PubMed ID
28752467
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