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Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency
Alisha M Gruntman1, Terence R Flotte2,3,4
1Horae Gene Therapy Center, University of Massachusetts Medical School, Suite 340, 55 Lake Avenue North, Worcester, MA, 01655, USA.
Methods in Molecular Biology (Clifton, N.J.)
|July 29, 2017
Summary
Alpha-1 antitrypsin deficiency (AATD) presents various phenotypes. This review covers current and future therapies, including genome editing, for AATD treatment.
Area of Science:
- Genetics and Medicine
- Pulmonology
- Hepatology
Background:
- Alpha-1 antitrypsin deficiency (AATD) is an inherited disorder.
- It can lead to lung and liver diseases.
- Genetic variations cause AATD.
Purpose of the Study:
- To provide an overview of AATD phenotypes.
- To describe current therapeutic strategies for AATD.
- To explore potential future treatments like genome editing.
Main Methods:
- Literature review of AATD.
- Analysis of existing and emerging therapies.
- Discussion of novel treatment modalities.
Main Results:
- AATD has diverse clinical presentations.
- Current therapies aim to manage symptoms and replace AAT.
- Genome editing shows promise for a definitive cure.
Conclusions:
- Understanding AATD phenotypes is crucial for tailored treatment.
- A combination of current and future therapies may offer comprehensive AATD management.
- Genome editing represents a potential paradigm shift in AATD treatment.
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