There's More Than One Way To Cure Diseases With Genes

A recent roundup in The Economist looks at a few new ideas for gene therapy.

DNA

Oak Ridge National Laboratory

Many people have inherited genes that are making them sick, or will, one day. Troublesome genes are part and parcel of being human. In the future, however, doctors may have some fixes, ranging from giving their patients working genes on top of their faulty DNA, to repairing the faulty DNA itself. The Economist recently published an overview of different approaches to genetic engineering that's worth a read.

Gene therapy usually aims to reverse heritable diseases by adding working versions of the genes that cause the diseases. It doesn't always function as promised: In early gene-therapy experiments, people's bodies sometimes mounted fatal immune responses to their therapies. Recently, however, scientists have seen some promising early results in gene therapies for choroideremia and Leber's congenital amaurosis, two inherited diseases that cause blindness. The Economist goes over those, but also summarizes a few other ideas researchers have for curing illness with genes.

One is using gene therapy as a cancer treatment. Instead of adding "good" copies of patients' "bad" genes, the therapy soups up patients' own immune cells with genes that help the cells target the patient's tumor._ The Economist_ highlights the work of Michel Sadelain of the Memorial Sloan-Kettering Cancer Center in New York, who is trying to treat leukemias and lymphomas with gene therapy.

Another group, led by Sha Jiahao of Nanjing Medical University in China, has found a way to alter the DNA in monkeys, a feat that's a first step toward repairing DNA in people.

Read the full story at The Economist.