Back to results
US 2024/0270822 A1Published application
Platelet targeted treatment
AssigneePlatelet Targeted Therapeutics, LLC
InventorsDavid A. Wilcox, Sandra L. Haberichter
Life of this patent
Estimating the term…
Abstract
The present disclosure relates to compositions and methods for targeting expression of exogenous genes to platelets. In particular, the present disclosure relates to treatment of hemophilia and other diseases and conditions by targeting expression of exogenous agents (e.g.. clotting factors) to platelets.
Claims20
1-16. (canceled)
A method of treating Glanzmann Thrombasthenia, comprising:
- contacting a hematopoietic stem cell with a composition comprising an expression vector comprising a) an expression cassette comprising a fragment of the integrin αIIb gene (ITGA2B) promoter;
- and an exogenous glycoprotein gene selected from the group consisting of glycoprotein IIb and glycoprotein Illa operably linked to said expression cassette to generate a modified stem cell under conditions such that said glycoprotein gene is expressed in said modified stem cell.
Dependencies are inferred from the claim text.