Integrin alphaIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus-transduced human hematopoietic cells.  (Wilcox DA, Olsen JC, Ishizawa L, Griffith M, White GC 2nd) Proc Natl Acad Sci USA 1999 Aug 17;96(17):9654-9.

Induction of megakaryocytes to synthesize and store a releasable pool of human factor VIII.  (Wilcox DA, Shi Q, Nurden P, Haberichter SL, Rosenberg JB, Johnson BD, Nurden AT, White GC 2nd, Montgomery RR) J Thromb Haemost. 2003 Dec;1(12):2477-89.

Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity.  Kuether EL(1), Schroeder JA, Fahs SA, Cooley BC, Chen Y, Montgomery RR, Wilcox DA, Shi Q.  J Thromb Haemost. 2012 Aug;10(8):1570-80. doi: 10.1111/j.1538-7836.2012.04791.x.  Comment in J Thromb Haemost. 2012 Aug;10(8):1566-9.

Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A.  Du LM(1), Nurden P, Nurden AT, Nichols TC, Bellinger DA, Jensen ES, Haberichter SL, Merricks E, Raymer RA, Fang J, Koukouritaki SB, Jacobi PM, Hawkins TB, Cornetta K, Shi Q, Wilcox DA.  Nat Commun. 2013;4:2773.

Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A.  Shi Q, Wilcox DA, Fahs SA, Fang J, Johnson BD, DU LM, Desai D, Montgomery RR.  J Thromb Haemost. 2007 Feb;5(2):352-61.

 Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies.  Shi Q(1), Wilcox DA, Fahs SA, Weiler H, Wells CW, Cooley BC, Desai D, Morateck PA, Gorski J, Montgomery RR.  J Clin Invest. 2006 Jul;116(7):1974-82.

Platelet-Targeted FVIII “Pleightlet™” LV-HSC for Severe Hemophilia A: Pre-Clinical Research Supporting a Clinical Protocol for a First-In‑Human Trial   Wilcox, D.A., Armant, M.A., Du, L.M., Johnson, B.D., Bushman, F., Jobe, S., Shi, Q., Malec, L., Hari, P. (Poster Abstract Submission) National Hemophilia Foundation Annual Bleeding Disorders Conference, Virtual (08/25/21). Poster 38 Accepted.



Integrin αIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus-transduced human hematopoietic cells.  Wilcox DA, Olsen JC, Ishizawa L, Griffith M, White GC. Proc. Natl. Acad. Sci. USA Vol 96, pp 9654-9659, August 1999, Cell Biology.

Megakaryocyte-targeted synthesis of the integrin beta(3)-subunit results in the phenotypic correction of Glanzmann thrombasthenia. (Wilcox DA, Olsen JC, Ishizawa L, Bray PF, French DL, Steeber DA, Bell WR, Griffith M, White GC 2nd) Blood. 2000 Jun 15;95(12).

Gene therapy for platelet disorders: studies with Glanzmann’s thrombasthenia.  Wilcox DA(1), White GC 2nd. J Thromb Haemost. 2003 Nov;1(11):2300-11.

Therapeutic expression of the platelet-specific integrin, alphaIIbbeta3, in a murine model for Glanzmann thrombasthenia.  Fang J, Hodivala-Dilke K, Johnson BD, Du LM, Hynes RO, White GC 2nd, Wilcox DA.  Blood. 2005 Oct 15;106(8):2671-9. Epub 2005 Jun 21.

Platelet gene therapy improves hemostatic function for integrin alphaIIb beta3-deficient dogs.  Fang J, Jensen ES, Boudreaux MK, Du LM, Hawkins TB, Koukouritaki SB, Cornetta K, Wilcox DA.  Proc Natl Acad Sci U S A. 2011 Jun 7;108(23):9583-8. doi: 10.1073/pnas.1016394108. Epub 2011 May 23.

Glanzmann thrombasthenia: state of the art and future directions.  Nurden AT(1), Pillois X, Wilcox DA. Semin Thromb Hemost. 2013 Sep;39(6):642-55. doi: 10.1055/s-0033-1353393. Epub 2013 Aug 8.



Platelets engineered to store interleukin-24 inhibited melanoma growth in mice.  Fang J, Yao M, Jing W, Sun B, Johnson BD, Wilcox DA.  2015 International Society on Thrombosis and Haemostasis 13 (Suppl. 2) (2015) 1–997.

Platelets Engineered to Express Interleukin-24 Inhibited Melanoma Tumor Growth in Mice.  Fang J, Yao M, Jing W, Sun B, Johnson BD, Wilcox DA.  ASGCT 19th Annual Meeting, May 4-7, 2016, Washington DC

Megakaryocyte- and megakaryocyte precursor-related gene therapies.  Wilcox DA Blood. 2016 Mar 10;127(10):1260-8. doi: 10.1182/blood-2015-07-607937. Epub 2016 Jan 19.




Platelet Targeted Treatment – Intellectual Property

South Africa Patent 2015/02945 issued 07-Sep-2016

Republic of Korea Patent 10-1819803 issued 11-Jan-2018

USA Patent 9,982,034 issued 29-May-2018

USA Patent 10,294,291 issued 21-May-2019

Israel Patent 238417 issued 19-Jul-2019

Canada Patent 2888982 issued 21-Jul-2020

Europe Patent 2912186 issued 06-Jan-2021 – 2021 01 06 European Divisional Validation Certificates Issued: Belgium, France, Germany, Luxembourg, Switzerland/Liechtenstein, United Kingdom, Denmark, Sweden, Austria, Italy, Poland, and Spain

Brazil Patent BR1120150092292, issued 15-June-2021

Hong Kong Patent No 1211986 issued 30-April-2021

India Patent 377388 issued 21 Sep 2021

Japan Patent 6975105 issued 9-Nov-2021