MEMPHIS, Tenn. (localmemphis.com) – “This is the first molecular disease, and we can’t do anything to help it,” says Dr. Mitchell Weiss. Dr. Weiss is talking about Sickle Cell Anemia. He specializes in pediatric hematology and oncology at St. Jude Children’s Research Hospital.
Sickle Cell Anemia affects more than 4,000 people in The BluffCity. While there’s no cure, researchers at St. Jude are getting closer andcloser.
Dr. Weiss said, “We’re going from a situation where there’snot very much that we can do, to having a tremendous amount ofopportunities.”
Researchers have found a way to use gene-editing therapy calledCRISPR to essentially fix sickle cell disease. CRISPR is short for ‘clusteredregularly interspaced palindromic repeats.’ and can be used to “edit”the DNA of living human cells.
Dr. Shengdar Tsai works in experimental hematology at St. Jude.
He said, “It’s basically like a small molecular scissor, thatcan cut the DNA at specific points, and the cell could repair the DNA to makeparticular changes.”
Those changes could cure a Sickle Cell patient.
Here’s how it works: Dr. Tsai just mentioned that molecularscissor; the “scissor,” or enzyme, cuts the bad DNA inside acell. This creates a mutation that fixes the DNA. As cells grow anddivide, as they do in our body at any given moment, newly “edited”cells are formed.
While it seems like the next best thing, critics in the medicalfield say that such a mutation can create more problems later, possibly causingother health issues.
While the tools and technology are here, this therapy is not readyto be mass-produced. This is the experimental phase.
Dr. Weiss said, “What we would like to see is for thisresearch to reach a point where it becomes approved as a standard care.”