Stanford University School of Medicine researcher Matthew Porteus, MD, PhD, was one of three expert witnesses who spoke at a U.S. Senate committee hearing on the CRISPR gene editing technology this week.
The hearing, held by the Senate Health, Education, Labor and Pensions Committee, was intended as an information-gathering event about the relatively new genetic technology. Senator Lamar Alexander, R-Tenn., said he got the idea for holding the hearing when he was fishing in Alaska and listening to the radio for the weather forecast when he heard a story about CRISPR. "I took notes and was fascinated, and I thought, 'one of the privileges of being chairman of this committee is that I can have a hearing on that,'”"Alexander said. "While CRISPR and other gene editing technologies could transform human health, it's not hard to see how we can quickly get into societal and ethical issues."
The witnesses, who included Katrine Bosley, CEO and president of Editas Medicine, and Jeffrey Kahn, MD, PhD, director of the Berman Institute of Bioethics at Johns Hopkins Medicine, described the technology to the senators and fielded questions.
“[CRISPR] gives us a method to correct typographical errors in the DNA,” Porteus said. “Tens of millions of people in the U.S. and hundreds of millions around the world could benefit from this technology.”
Porteus outlined his plans to use gene editing to correct the genetic defect that causes sickle cell anemia. He described how researchers hope to launch a clinical trial that would remove blood-forming stem cells from a patient, use CRISPR technology to alter the mutation that causes sickle cell disease, perform checks to ensure the cells are otherwise healthy, and then reintroduce them into the body.
"That's amazing," said Sen. Tim Scott, R-S.C. "That's pretty cool."
The senators asked if there should be any new legislation or changes in the regulation of CRISPR and other gene-editing technologies. Porteus and the other witnesses said no, that existing regulatory structure was adequate for overseeing the safety of the technology in research and upcoming clinical trials. Porteus said that the best way to move forward was to not create any additional regulation and to “allow a thousand trees to grow, because one of these may hold the answer we are looking for.”
Porteus also talked about using CRISPR to beat antibiotic resistant bacteria and cure many other diseases that are currently considered to be incurable. He said the work that is currently being done would be a platform that is adaptable to address many different diseases.
Previously: Use caution when editing genes, new report advices, Stanford scientists describe stem-cell and gene-therapy advances in scientific symposium and A step closer to gene therapy for sickle cell disease
Photo by MarkThomas