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RCT Patents Versatile Gene Expression Enhancer

5.23.2001

Tucson, AZ – May 23, 2001

U.S. Patent No. 6,225,082 issued recently to Research Corporation Technologies (RCT) for a gene expression technology that enhances protein expression in mammalian cells.

The RNA translation enhancer (RTE) sequence, derived from an untranslated region of the myelin basic protein messenger RNA (mRNA), works two ways. It increases the rate of mRNA transported from the cell nucleus and boosts activity at the associated ribosomes.

This sequence may be useful in human gene therapies to increase protein production of therapeutic genes. It may also help increase production of recombinant proteins in culture and in transgenic plants. The new patent also protects use of the RTE sequence in a DNA vector and with reporter genes.

The RTE sequence is one of several gene expression components available through RCT’s Gene Expression Technologies (GET) licensing program, which offers gene expression systems and basic technologies at reasonable rates. RCT manages this technology for the University of Connecticut and its researchers who discovered the sequence, Drs. John H. Carson, Sunjong Kwon, Devin Aigner and Daniela Avossa.

Knowing that the RTE sequence is small and unrelated to gene transcription, Carson and colleagues wondered how it would act outside its natural environment. They constructed vectors and tested the sequence with a variety of cell types. Their results showed the RTE sequence can be a general enhancer for heterologous gene expression.

The key to gene therapy’s success will depend on the amount of protein expressed by the different vector systems. Current technologies for enhancing gene expression target the transcriptional level, but are unsuitable for regulated or tissue-specific expression. Use of translation enhancers that affect protein synthesis at the ribosomes have so far been unsuccessful in gene therapies. Other types of enhancers are inefficient or specific to particular genes.

Since the RTE sequence is a general enhancer, it could help increase protein expression levels in a variety of gene therapies for sickle cell anemia, cancer, genetic diseases and other indications. This technology may complement the current work of all the major gene therapy companies.

Contact

For more information about this technology and the GET program, contact Bennett N. Cohen, Ph.D., Director, (520) 748-4400 or fax (520) 748-0025.