That foresight culminated in the design and present construction of a highly specialized, ultra-clean environment for the production of experimental biological therapeutics for initial phases of human clinical trials. Over the past twenty years, breakthroughs in molecular biology and genetics have profoundly advanced our understanding of the genetic basis of numerous diseases. Gene therapy has the potential to treat the over 6,000 disorders that can be traced to an anomaly in the underlying human genome. Scientists have developed techniques for isolating and characterizing nonfunctioning gene and successfully transplanting a functioning or therapeutic gene into human cells. The key to successful gene therapy is identifying the most efficient means of delivering therapeutic genes.
Vectors are vehicles into which healthy genes can be packaged and carried into human cells.
Initially, in order to get DNA into as many healthy cells as possible, the infectious nature of viruses was viewed as an advantage. It remains a complicated procedure to maintain the helpful viral properties that enable the vector to infect a high number of cells and also allow sufficient space inside the body of the virus for inserting genetic materials. Moreover, viral gene delivery systems continue to trigger an immune response that limits their efficacy. Non-viral gene therapy agents present a promising alternative to the immunological problems posed by viruses. Waisman Center scientists hope to discover cures for neuromuscular, metabolic and neurodegenerative diseases through the development of novel gene therapy techniques.