May 15, 2025

Federally funded scientific research is vital for developing new medical treatments such as the gene-editing treatment described in this NYT article, especially for rare diseases and cancers. Drastic cuts in research funding will slow the discovery and development of new treatments. Please remember this when you contact or vote for your Congressional representatives.

 

"More than 30 million people in the United States have one of more than 7,000 rare genetic diseases. Most are so rare that no company is willing to spend years developing a gene therapy that so few people would need.

 

But KJ’s treatment — which built on decades of federally funded research — offers a new path for companies to develop personalized treatments without going through years of expensive development and testing."

 

"It eventually could also be used for more common genetic disorders like sickle cell disease, cystic fibrosis, Huntington’s disease and muscular dystrophy."

 

"The researchers emphasized the role government funding played in the development.

 

The work, they said, began decades ago with federal funding for basic research on bacterial immune systems. That led eventually, with more federal support, to the discovery of CRISPR. Federal investment in sequencing the human genome made it possible to identify KJ’s mutation. U.S. funding supported Dr. Liu’s lab and its editing discovery. A federal program to study gene editing supported Dr. Musunuru’s research. Going along in parallel was federally funded work that led to an understanding of KJ’s disease."