A little boy, born in Philadelphia in 2024 with a potentially fatal genetic deficiency, became the first person treated with personalized gene edition therapy. Doctors used CRISPR technology to quickly create a personalized treatment for KJ, targeting the specific mutation of the child. The boy responds to development milestones, his weight is in the 40th centile, but his need for liver transplant is still unknown, according to reports. This medical breakthrough could revolutionize treatment for children with rare genetic diseases that need intervention in months rather than in years.
In its cover, the New York Times sharp references The role of federal science financing in this advance:
The treatment of KJ – which has been built on decades of research funded by the federal government – offers a new path to companies to develop personalized treatments without going through years of development and expensive tests. . . The researchers highlighted the role that government financing played in development. The work, they said, started decades ago with federal funding for basic research on bacterial immune systems. This finally led, with more federal support, to the discovery of CRISPR. The federal investment in the sequencing of the human genome made it possible to identify the transfer of KJ. US funding supported Dr. Liu’s laboratory and its publishing discovery. A federal program to study the edition of genes supported Dr Musunuru’s research. The parallel was funded by the federal government which led to an understanding of KJ’s disease. “I don’t think it could have happened in a country other than in the United States,” said Dr. Urnov.
This powerful example of the advantages of scientific research funded by the federal government is something that Washington decision -makers should keep in mind given the current republican inclination To cut such an investment. (And here are some other examples.))
They may also want to keep the new Nber working document in mind, “Social returns to public R&D“, In which economists Andrew J. Fieldhouse and Karel Mertens find that federal investments in R&D are a powerful engine of economic growth and prosperity. Such expenses have remarkably high of social yields, ranging from 140 percent to 210% for public returns from the public which are not considerably widespread by the private industries.
According to the article: “If R&D increases under the law on fleas and sciences has been fully appropriate, our modeling indicates an increase in American productivity in a few years, reaching gains from 0.2 to 0.4% after seven years or more.”
Unfortunately, federal R&D expenses have decreased the summits of the era of the Cold War by 1.8% of GDP only 0.75% today. And recent efforts suggest that Washington could go further in the wrong direction. (In my 2023 book, The conservative futuristic: how to create the science fiction world that was promised to usI suggest returning to these levels of funding for the Cold War / Spatial Age, as well as some key expenses reforms.)
Allow me to finish with a quote from the Financial Times of a genetics teacher on the treatment of the child of Philadelphia: “It seems to me to be a scientific” miracle “which made it possible to cure a very rare serious illness and provides knowledge to deal with many other diseases.”