Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency

Alisha M Gruntman; Terence R Flotte

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

Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency

Methods Mol Biol. 2017;1639:267-275. doi: 10.1007/978-1-4939-7163-3_27.

Authors

Alisha M Gruntman¹, Terence R Flotte^{2

3

4}

Affiliations

¹ Horae Gene Therapy Center, University of Massachusetts Medical School, Suite 340, 55 Lake Avenue North, Worcester, MA, 01655, USA.
² Horae Gene Therapy Center, University of Massachusetts Medical School, Suite 340, 55 Lake Avenue North, Worcester, MA, 01655, USA. terry.flotte@umassmed.edu.
³ Department of Pediatrics, University of Massachusetts Medical School, Suite 340, 55 Lake Avenue North, Worcester, MA, 01655, USA. terry.flotte@umassmed.edu.
⁴ Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Suite 340, 55 Lake Avenue North, Worcester, MA, 01655, USA. terry.flotte@umassmed.edu.

PMID: 28752467
DOI: 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.

Keywords: CRISPR/Cas9; Emphysema; Genome editing; Liver disease; SERPINA1.

MeSH terms

Gene Editing
Genetic Therapy*
Humans
Liver Diseases / genetics
Liver Diseases / therapy
Lung Diseases / genetics
Lung Diseases / therapy
alpha 1-Antitrypsin / genetics
alpha 1-Antitrypsin / therapeutic use
alpha 1-Antitrypsin Deficiency / genetics*
alpha 1-Antitrypsin Deficiency / therapy*

Substances

alpha 1-Antitrypsin