Improved clinical parameters by expression of factor viii
AssigneeSangamo Therapeutics, Inc.
InventorsEdward R. Conner, Brigit E. Riley, Didier Rouy
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Abstract
Described herein are constructs used for liver-specific expression of a transgene.
Claims20
A method of increasing Factor VIII (FVIII) protein in a human subject, comprising administering to the human subject one or more doses of 5×1012 to 5×1013 vg/kg of an Adenovirus-Associated Virus (AAV) vector that encodes a FVIII protein, wherein administration of the AAV vector results in a clinically relevant increase in the level of circulating FVIII activity.
A method of increasing Factor VIII (FVIII) protein in a human subject, comprising administering to the human subject one or more doses of 5×1012 to 5×1013 vg/kg of an Adenovirus-Associated Virus (AAV) vector that encodes a FVIII protein, wherein administration of the AAV vector results in a reduction of the number of FVIII treatments the human subject undergoes.
Dependencies are inferred from the claim text.