THERAPEUTIC CLASS

SERPINA1 mRNA as a Treatment for Alpha-1 Antitrypsin Deficiency

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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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Detail Information

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Language

English

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Classification

NONE

Content Type

LITERATURE

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Subject(s)

Respiratory system
RHINOS
HISTRINE
Deslotine
Modexa
Ritez
Histrine Levo

Specific Detail Info

Hindawi Journal of Nucleic Acids Volume 2018,

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