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Research Matters: Strides in Gene Therapy Initiated by Minnesota Sea Grant Research

Minnesota Sea Grant researchers' work with fish genetics has led to revolutionary advances in human gene therapy.

In 1997, Minnesota Sea Grant funded a project that aimed to engineer faster-growing fish for aquaculture purposes. This project led researchers to discover and transform an inactive gene originating in the salmon family into a non-viral DNA delivery system called the Sleeping Beauty (SB) transposon system. This system can transport normal genes into genetically damaged cells that cause such things as retinal degeneration and cancers in humans.

Since the 1980s, Minnesota Sea Grant has actively provided a financial base for University of Minnesota researchers, such as Dr. Perry Hackett, to explore the genetics of economically important fish. This initial support from Minnesota Sea Grant has since spurred private sector interest and more than $5 million in direct funding to support further research in SB's application to human gene therapy.

Since its creation, SB has inspired the founding of Discovery Genomics, Inc. by Minnesota Sea Grant researchers and made significant advancements in human gene therapy through laboratory experiments on mice. Recently, researchers at the University of Texas MD Anderson Cancer Center used SB to perform clinical trials on human patients with blood disorders including lymphocytic leukemia and non-Hodgkin lymphoma. No severe or long-term toxicity was found among any of the 33 patients, and 47% of patients treated remained in complete remission or showed disease regression.

By Sea Grant Staff
June 2015

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