Margaret Goodell, Ph.D. Banking on Bone Marrow

MDA Quest Magazine | November 2005

“It’s a way to get at virtually all of the muscles in the body, not just the major ones that we can see. Every muscle fiber is fed by the bloodstream in one way or another, so if you can really get something delivered through the bloodstream rather than in some localized way, it’s potentially a very powerful therapy.”  read story

Running a Stop Sign

MDA Quest Magazine | April 2005

PTC124 sticks to ribosomes — the cells’ protein factories — and prompts them to interpret a premature stop codon as a normal codon. Instead of aborting assembly of the protein, the ribosome inserts a protein building block — an amino acid — and continues making a complete protein chain until it encounters the normal termination codon, which the ribosome correctly interprets as a stop. read story