Gruntman Alisha M, Flotte Terence R
Horae Gene Therapy Center, University of Massachusetts Medical School, Suite 340, 55 Lake Avenue North, Worcester, MA, 01655, USA.
Department of Pediatrics, University of Massachusetts Medical School, Suite 340, 55 Lake Avenue North, Worcester, MA, 01655, USA.
Methods Mol Biol. 2017;1639:267-275. doi: 10.1007/978-1-4939-7163-3_27.
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.
本综述旨在概述α-1抗胰蛋白酶缺乏症，包括其所涵盖的不同疾病表型。然后，我们描述了为应对这些不同表型而进行的各种治疗尝试。最后，我们讨论了未来潜在的治疗方法，如基因组编辑，以及它们在治疗α-1抗胰蛋白酶缺乏症中可能发挥的作用。
