Safety test for gene therapy vectors (SJ-15-0027)

St. Jude Reference #SJ-15-0027


Gene therapy vectors hold promise to revolutionize treatment for many diseases, but also carry a risk of causing cancer if they integrate into oncogenic sites such as LMO2. 

Researchers at St. Jude have developed a cell based assay that can be used to assess the relative risk that any given gene therapy vector will integrate into and consequently activate Lmo2 and other T-cell proto-oncogenes.   This assay was able to detect the oncogenic activity of the clinical MFG-rc vector that caused 5 cases of leukemia in an early SCID-X1 gene therapy trial.  This assay can be used to compare the relative oncogenic potential of a variety of gamma-RV and lentiviral vectors and to assess the risk conferred by various transcriptional elements contained in these genomes. 

Use of this assay can help improve the safety of integrative gene therapy vectors for diseases such as sickle cell anemia, immunodeficiency, etc. in a relatively easy yet sensitive and clinically relevant way.


SCID-X1 gene therapy trial, leukemia

Granted patents or published applications

U.S. Patent No. 11,268,158 issued March 8, 2022, “Assay for safety assessment of therapeutic genetic manipulations, gene therapy vectors and compounds."

Related scientific references

Sheng Zhou, Soghra Fatima, Zhijun Ma, Yong-Dong Wang, Taihe Lu, Laura J Janke, Yang Du and Brian P Sorrentino, “Evaluating the Safety of Retroviral Vectors Based on Insertional Oncogene Activation and Blocked Differentiation in Cultured Thymocytes,” MTOpen, Received 6 January 2016; accepted 24 February 2016; advance online publication 12 April 2016. doi:10.1038/mt.2016.55

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