The Conversation on Biotechnology by Marc Zimmer

Author:Marc Zimmer
Language: eng
Format: epub
Publisher: Johns Hopkins University Press
Published: 2023-06-15T00:00:00+00:00

The Rise of Gene Therapies

Thousands of diseases are the result of DNA errors, which prevent cells from functioning normally.1 Currently, only 5 percent of the roughly 7,000 rare diseases have an FDA-approved drug, leaving thousands of conditions without a pharmaceutical treatment. But over the past few years, genetic engineering technology has made impressive strides toward the ultimate goal of curing disease by changing a cell’s genetic instructions. By directly correcting disease-causing mutations or altering a cell’s DNA to give the cell new tools to fight disease, gene therapy offers a powerful new approach to medicine. The resulting gene therapies will be able to treat many diseases at the DNA level in a single dose.

In 2022 there were more than 2,000 gene therapies in development around the world. A large fraction of this research focuses on rare genetic diseases, which affect 400 million people worldwide. We may soon see cures for rare diseases like sickle cell disease,2 muscular dystrophy,3 and progeria, a rare and progressive genetic disorder that causes children to age rapidly. Farther into the future, gene therapies may treat more common conditions, like heart disease and chronic pain.

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