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New Therapeutic Targets for Alpha-1 Antitrypsin Deficiency

Lomas, DA; (2018) New Therapeutic Targets for Alpha-1 Antitrypsin Deficiency. Chronic Obstructive Pulmonary Diseases , 5 (4) pp. 233-243. 10.15326/jcopdf.5.4.2017.0165.

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Alpha-1antitrypsin deficiency (AATD) results from the intracellular polymerization and retention of mutant alpha-1antitrypsin (AAT) within the endoplasmic reticulum of hepatocytes. This causes cirrhosis whilst the deficiency of circulating AAT predisposes to early onset emphysema. This is an exciting time for researchers in the field with the development of novel therapies based on understanding the pathobiology of disease. I review here augmentation therapy to prevent the progression of lung disease and a range of approaches to treat the liver disease associated with the accumulation of mutant AAT: modifying proteostasis networks that are activated by Z AAT polymers, stimulating autophagy, small interfering RNA and small molecules to block intracellular polymerization, and stem cell technology to correct the genetic defect that underlies AATD.

Type: Article
Title: New Therapeutic Targets for Alpha-1 Antitrypsin Deficiency
DOI: 10.15326/jcopdf.5.4.2017.0165
Publisher version: https://doi.org/10.15326/jcopdf.5.4.2017.0165
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: emphysema, alpha-1 antitrypsin deficiency, cirrhosis, conformational disease, small molecules, siRNA, proteostasis, autophagy
UCL classification: UCL
UCL > Provost and Vice Provost Offices > VP: Health
URI: https://discovery.ucl.ac.uk/id/eprint/10064813
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