Glanzmann's thrombasthenia future or investigational therapies

Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]

Overview

Gene therapy is a cutting the edge method for treatment of various diseases. Gene therapy in animal models demonstrates good potential of this treatment for curing patients with Glanzmann’s thrombasthenia. As an example for targeting transgene expression in the developing megakaryocytes from two patients with GT in vitro, αIIbβ3 promoter was expressed on these transduced megakaryocytes. CD34+ cells was demonstrated utilizing mice leukemia retrovirus vectors, by this method the Glanzmann’s thrombasthenia was corrected.^[1]

Future or Investigational Therapy

Gene therapy has a significant potential for curing Glanzmann’s thrombasthenia in the near future.^[2]

References

↑ Blickstein D, Dardik R, Rosenthal E, Lahav J, Molad Y, Inbal A (2014). “Acquired thrombasthenia due to inhibitory effect of glycoprotein IIbIIIa autoantibodies”. Isr Med Assoc J. 16 (5): 307–10. PMID 24979837.
↑ Solh T, Botsford A, Solh M (2015). “Glanzmann’s thrombasthenia: pathogenesis, diagnosis, and current and emerging treatment options”. J Blood Med. 6: 219–27. doi:10.2147/JBM.S71319. PMC 4501245. PMID 26185478.

[pmid24979837-1] Blickstein D, Dardik R, Rosenthal E, Lahav J, Molad Y, Inbal A (2014). “Acquired thrombasthenia due to inhibitory effect of glycoprotein IIbIIIa autoantibodies”. Isr Med Assoc J. 16 (5): 307–10. PMID 24979837.

[pmid26185478-2] Solh T, Botsford A, Solh M (2015). “Glanzmann’s thrombasthenia: pathogenesis, diagnosis, and current and emerging treatment options”. J Blood Med. 6: 219–27. doi:10.2147/JBM.S71319. PMC 4501245. PMID 26185478.

[1]

[2]