MRNA THERAPY FOR UREA CYCLE DISORDERS
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United States of America Patent
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Issued Date -
Oct 2, 2014
app pub date -
Jun 19, 2014
filing date -
Dec 1, 2009
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Abstract
Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.
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