How the U.S. government helped fund the development of gene therapy

Over at Discover magazine, Veronique Greenwood has written a terrific article explaining the development of gene therapy, one of the most promising avenues of medical research over the last several decades. And, she explains, this basic, life-saving research couldn't have been done without the help of government funding:

After years of doubts and dangers, gene therapy is showing promise as one of our best hopes for fighting lethal and debilitating diseases (see the DISCOVER feature "The Second Coming of Gene Therapy"). In the lab, replacing malfunctioning genes with working ones has succeeded in curing or treating animals with hemophilia, (the animal analogs of) depression, and other afflictions. In clinical trials in people, it's brought sight to the blind, allowed kids without immune systems to live normally, and contributed to one of the most talked-about cancer discoveries of the year, a promising experimental treatment for leukemia.

Of course, the technique was not brought to us from above by Prometheus-it all rests on a mountain of basic science research, most of it publicly funded.

Greenwood goes on to offer a detailed timeline of all the painstaking research over a half century that led to today's successful (though still experimental) gene therapy treatments. At every stage, these medical breakthroughs wouldn't have been possible without public funding.

Here are some highlights of that timeline:

1952: Hershey and Chase show that DNA is the genetic material, bearing heritable traits from generation to generation. Most scientists previously thought the leading contender for that role was proteins, though experiments in 1944 had suggested DNA was more likely. Hershey goes on to win the Nobel Prize.

Funded in part by the NIH.

. . .

1966: Edward Tatum suggests in a speech that genetic disease might be treated with "genetic engineering." He suggests that nonpathogenic viruses be used to transfer genes to patients' cells, as researchers had previously observed that viruses could, at least temporarily, introduce DNA into bacterial and mammalian cells.
In the same year, Joshua Lederberg, who had mentioned the idea of gene therapy as early as 1963, wrote an article for The American Naturalist describing how it might be done, describing it as "virogenic therapy." First, however, researchers needed a method for isolating the gene they'd like to insert.

1969: Jonathan Beckwith at Harvard and colleagues are the first to purify a gene, using viruses to selectively snap up the proper gene from a soup of DNA from pureed E. coli. Once researchers learn to isolate genes, though, they still have to figure out how to introduce them into viruses.

Funded in part by the NIH and the NSF.

. . .

1990: The first FDA-approved gene therapy trial takes place at the NIH, when two girls with a type of severe combined immunodeficiency syndrome (SCID) caused by an enzyme deficiency are infused with modified versions of their own blood cells that carries genes for the functioning version of the enzyme. The effects are temporary but observable. For the long term, the patients still have to receive enzyme injections to treat their disease.

Funded by the NIH.

. . .

2011: Another success makes headlines: David Porter and Carl June report that immune cells modified with gene therapy had cured two terminal leukemia patients of their cancer. Though the treatment is still in the very early stages, its striking results, along with the success of the blindness study two years prior, bring new attention to gene therapy's potential.

The National Cancer Institute is now funding a study to determine what specific characteristics led to that trial's success, comparing two sets of modified immune cells in patients. Porter, June, and their collaborators are currently recruiting patients.

Funded in part by the NIH.

You can read the rest of the article at Discover Magazine.

How the U.S. government helped fund the development of gene therapy

This article is part of the Public Science Triumphs series, which celebrates the ways that publicly-funded science has enriched our lives and paved the way for a better future.

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