Image of SERPINA1 mRNA as a Treatment for Alpha-1 Antitrypsin Deficiency

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SERPINA1 mRNA as a Treatment for Alpha-1 Antitrypsin Deficiency


Alpha-1-antitrypsin (AAT) deficiency is a genetic disorder that produces inactive/defective AAT due tomutations in the SERPINA1
gene encoding AAT. This disease is associated with decreased activity of AAT in the lungs and deposition of excessive defective
AAT protein in the liver. Currently there is no specific treatment for liver disease associated with AAT deficiency. AAT lung disease
is often treated with one of several serum protein replacement products; however, long-termstudies of the effectiveness of SerpinA1 replacement therapy are not available, and it does not reduce liver damage inAAT deficiency.mRNAtherapy could potentially target both the liver and lungs of AAT deficient patients.


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English
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NONE
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Hindawi Journal of Nucleic Acids Volume 2018,
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