Hemophilia future or investigational therapies

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1];Associate Editor(s)-in-Chief: Sabawoon Mirwais, M.B.B.S, M.D.[2]

Overview

Future and investigational therapies of hemophilia include co-transplantation of endothelial colony-forming cells (ECFCs) and placenta-derived mesenchymal stromal cells (PMSCs) at the neonatal age and prevention of the anti-factor VIII memory B-cell response.

Future and Investigational Therapies

Future and investigational therapies of hemophilia include:

Co-transplantation of endothelial colony-forming cells (ECFCs) and placenta-derived mesenchymal stromal cells (PMSCs)

  • This is done at the neonatal age.[1]
  • It is a potential strategy to achieve stable and long-term engraftment.[1]
  • This holds great promise for the cell-based treatment of hemophilia A.

Prevention of the anti-factor VIII memory B-cell response

References

  1. 1.0 1.1 Gao K, Kumar P, Cortez-Toledo E, Hao D, Reynaga L, Rose M, Wang C, Farmer D, Nolta J, Zhou J, Zhou P, Wang A (January 2019). "Potential long-term treatment of hemophilia A by neonatal co-transplantation of cord blood-derived endothelial colony-forming cells and placental mesenchymal stromal cells". Stem Cell Res Ther. 10 (1): 34. doi:10.1186/s13287-019-1138-8. PMC 6341603. PMID 30670078.
  2. Delignat S, Russick J, Gangadharan B, Rayes J, Ing M, Voorberg J, Kaveri SV, Lacroix-Desmazes S (December 2018). "Prevention of the anti-factor VIII memory B-cell response by inhibition of the Bruton's tyrosine kinase in experimental hemophilia A". Haematologica. doi:10.3324/haematol.2018.200279. PMID 30545924.

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