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Scientists investigate several techniques to achieve successful gene therapy

San Jose, Calif.--May 2, 2003--As the dismal status of the entire biotech market dissolves numerous potentially lucrative partnerships, many companies developing gene therapy are focusing their R&D efforts on only a few key products.

The idea is to have enough therapies in the pipeline to balance inevitable failures, but not so many that they drain the already strained financial resources.

"The primary difficulty in developing clinically useful gene therapies has been designing a delivery system that delivers sufficient quantities of therapeutic DNA into enough cells to be expressed at levels that affect the disease being treated," states Technical Insights Analyst Katherine Austin.

Other approaches work by stimulating an immune response, mediating specific cell killing, activating a pro-drug, or producing a molecular decoy required for the replication of a virus. DNA vaccines are also in clinical trials for various forms of cancer and infectious disease such as herpes, hepatitis, and AIDS.

The techniques currently vying for the position of successful gene therapy delivery system include viral vectors, ex vivo cell transfection, liposomal vectors, artificial chromosomes, matrix vectors, genetically engineered cells, gene activators, bacterial vectors, and naked DNA.

Research is on to investigate methods such as transcription factors, antisense inhibition or RNA interference, and gene repair to regulate expression without the transfer of genes. Vectors that target particular tissue and cell types to avoid the systemic exposure and side effects such as those seen in current chemotherapy protocols are also in the pipeline.

As vector systems demonstrate success in clinical trials and clinical applications for gene therapy are developed, the cancer, vascular diseases and cystic fibrosis markets are likely to be the first to reap financial benefits from gene therapy.

"Once the first successful products are on the
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Contact: Julia Paulson
jpaulson@frost.com
210-247-3870
Technical Insights
2-May-2003


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