CNRS. France: Research efforts associating scientists from the CNRS, UVSQ and INSERM within the Laboratoire END-ICAP1, working in collaboration with a team from the University of Bern, has demonstrated the therapeutic potential of a new class of synthetic oligonucleotides2 in the treatment of Duchenne muscular dystrophy (DMD) using RNA "surgery".
Tested in the mouse, this new generation of molecules proved to be clinically superior to those currently under evaluation in DMD patients, notably for restoring cardio-respiratory and central nervous system function. These findings were published on 2 February 2015 in Nature Medicine.