Optimised coding sequence and promoter
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United States of America Patent
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Jul 14, 2020
Grant Date -
Jan 11, 2018
app pub date -
Jul 17, 2017
filing date -
Jul 8, 2009
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Abstract
An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes arc alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.
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