Gene Therapy for Wiskott-Aldrich Syndrome (SJ-19-0012)

St. Jude Reference #SJ-19-0012


St. Jude Children’s Research Hospital is offering a stable cell line for producing a gene therapy vector for treating Wiskott-Aldrich Syndrome (WAS).The WAS vector uses the same lentiviral backbone as a vector made by St. Jude for treating X-linked severe combined immunodeficiency disease (“XSCID”), which is currently being tested in a phase I clinical trial.


Gene therapy, Wiskott, Aldrich, transplant, lentiviral

Granted Patents or Published Applications

Related Scientific References

Singh, S. et al., “Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector”, Molecular Therapy 4: 1-16 (Dec. 18, 2016). 

Licensing Opportunities

More information is available under a confidentiality agreement. Contact:

Contact the Office of Technology Licensing (Phone: 901-595-2342, Fax: 901-595-3148) for more information.