Thursday, October 11, 2012
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a-Antitrypsin (AAT) deficiency
deacetylase 7 (HDAC7)
In vitro studies suggest suberoylanilide
hydroxamic acid (SAHA) could help treat AAT deficiency.
The Z-variant of AAT deficiency is the most severe form of the condition,
which involves defective folding, trafficking and secretion of AAT from the
endoplasmic reticulum. In human cell lines expressing Z-AAT, HDAC
inhibitors including SAHA increased AAT trafficking from the endoplasmic
reticulum compared with vehicle. In these cells, small interfering RNA
knockdown of HDAC7 increased AAT stability and trafficking compared with
silencing of other HDACs. Next steps could include testing SAHA in animal
models of AAT deficiency.
At least six companies have therapeutics to treat AAT deficiency in
development stages ranging from preclinical to marketed.
& Co. Inc. markets Zolinza
SAHA to treat cutaneous T cell lymphoma (CTCL).
SciBX 5(40); doi:10.1038/scibx.2012.1051
Published online Oct. 11, 2012
Patent and licensing status
Bouchecareilh, M. et al.
J. Biol. Chem.; published online Sept. 20, 2012;
Contact: William E. Balch,
The Scripps Research Institute,
La Jolla, Calif.
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