The most important research strategies on HD are:
To Reduce the production of the pathologically changed protein (mHTT). Since the disease is caused by a single dominant gene that contains the code of the toxic protein, one strategy is to SILENCE this gene by inhibiting its activity, with sophisticated technologies and drugs called Anti Sense Oligonucleotides (ASO’s). These drugs stop the production of messenger RNA that, being related to the mutate gene, orders cells to produce mHTT. Trials are at the moment limited to animal models, but the cooperation between ISIS (that has expertise on ASO) and Roche (pharmaceutical legend) let us hope in we will start humans trials in the next years;
To Improve the ability of cells to survive to dangerous effects of mHTT, or do a stabilization with dopamine as to diminish symptoms. The more interesting molecule for this goal is pridopidine; there’s a trial of phase II in 29 centres in Europe and 22 in US;
To Use other strategies of ‘neural regeneration’ with cellular and molecular biology that protect neurons by interfering with inner workings of the disease.