Title

Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency

UMMS Affiliation

Horae Gene Therapy Center; Department of Pediatrics, Division of Pulmonary and Allergy

Date

7-28-2017

Document Type

Book Chapter

Disciplines

Genetics and Genomics | Molecular Biology | Therapeutics

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.

Rights and Permissions

Citation: Methods Mol Biol. 2017;1639:267-275. doi: 10.1007/978-1-4939-7163-3_27. Link to article on publisher's site

Related Resources

Link to Article in PubMed

Keywords

CRISPR/Cas9, Emphysema, Genome editing, Liver disease, SERPINA1

PubMed ID

28752467