Now, gene therapy features new tools and safer delivery system

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Gene therapy was once viewed as a promising new way to treat many incurable diseases — until a tragic death occurred during a clinical trial. It’s taken nearly two decades for the industry to recover, but now several Northeast Ohio researchers are once again developing DNA as medicine. This is the business of gene therapy.

It’s a technique developed in the late 1980’s that held great promise for treating patients with diseases like cystic fibrosis, Parkinson’s or rare blood disorders that had no other viable options.

Researchers recruit a virus to do what viruses do best — infect a cell with its DNA payload, like a rocket delivering supplies to the space station. But in gene therapy, that payload is engineered to repair whatever mutation was causing the disease.

After the tragedy in 1999, Bob Moen says Copernicus Therapeutics sidesteps the need for a virus to deliver the DNA by using DNA nanoparticles, which wraps the therapeutic gene with amino acids.

Read full, original post: After Three Decades, Gene Therapy Comes of Age

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