“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
Gene therapy, a high-tech approach that aims to fight diseases with genes rather than drugs, may one day become the chosen means for treating many medical disorders. Scientists seeking a gene therapy approach to ALS face some formidable hurdles but are making progress. read story
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