Genetics and Health

Genetic Medicine for Inherited Illnesses

by Hsien Hsien Lei, PhD on April 3rd, 2006

A new paper in Nature Reviews Genetics, entitled “Genetic Medicines: Treatment Strategies for Hereditary Disorders,” discusses potential genetic therapy for inherited diseases caused by a defect in one gene. (Not multifactorial diseases, such as cardiovascular disease, that are caused by a complex interaction of multiple genes and environmental factors.)

Genetic therapy techhniques discussed:

• Gene transfer using viral vectors, e.g., adenoviruses and retroviruses.
• RNA-modification therapies - suppressing or stimulating RNA that is the intermediate step between DNA and protein.
• Embryonic stem cells for regenerating organs, etc.

Dr. Timothy O’Connor, Assistant Research Professor of Genetic Medicine at Weill Cornell:

Remember, drug development is always a 10-to-15-year process, whatever the theory behind it. And just in the last decade we’ve seen enormous leaps forward, such as faster high-throughput screens, hapmap technologies and other advances. It’s our belief that even more astonishing advances are yet to come that will turn the dream of genetic medicine into a reality for patients at the bedside.

Not just at the bedside, but hopefully before. Preventive medicine is far more effective than medicine after people have fallen ill.

Medical News Today, April 3, 2006

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POSTED IN: Genetic Engineering