In utero transplantation of factor viii-expressing cells for treatment of hemophilia
AssigneesWake Forest University Health Sciences, Emory University, Children's Healthcare of Atlanta, Inc.
InventorsMaria Graca N.D. Almeida-Porada, Christopher D. Porada, Anthony Atala, Christopher B. Doering, H. Trent Spencer
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Abstract
Disclosed herein are method of treating hemophilia A in a subject comprising injecting the subject with mesenchymal stromal/stem cells (MSC) modified to express high levels of Factor VIII protein. The MSC are injected into the subject prenatally. The modified MSC may also express high levels von Willebrand factor protein.
Claims31
A method of treating a subject prenatally diagnosed as having hemophilia comprising injecting modified mesenchymal stem/stromal cells (MSC) into the subject in utero, wherein the modified MSC are modified to express high levels of Factor VIII protein or high levels of both Factor VIII protein and von Willebrand factor (vWF) protein.
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Dependencies are inferred from the claim text.