Title

Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency

UMMS Affiliation

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

Publication Date

2017-07-28

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.

Keywords

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

DOI of Published Version

10.1007/978-1-4939-7163-3_27

Source

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

Journal/Book/Conference Title

Methods in molecular biology (Clifton, N.J.)

Related Resources

Link to Article in PubMed

PubMed ID

28752467

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