- Category: Innovative care
- Published: Tuesday, 08 April 2014 11:02
Knowledge of the mechanisms of genetic diseases is indispensable to the development of new therapeutics. In effect, to know precisely which metabolic pathway is modified enables the definition of a therapeutic target, the obligatory first step in the search for new treatments.
Les biomCurrently, research is following numerous leads. In some cases, methods using techniques of “molecular screening” may identify a chemical or a biological molecule capable of bypassing a defect or restoring a deficient pathway. In other cases and depending on the type of gene mutation, research into new therapeutics may target the messenger RNA (notably, to avoid its degradation or to modify its composition) of the mutation involved.
When these approaches are not yet feasible, the replacement of defective or absent cells by the transplantation of normal cells (cell therapy) or the introduction into the defective cells (or their offsprings) of a normal copy of the mutated gene (gene therapy) constitute promising paths of research that will become even more important when it will be possible to use stem cells.
Thus, Imagine plans to transform knowledge on genetic diseases into effective treatments as rapidly as possible. We foresee that this research will also have an impact on more common diseases by serving as models for the development of their treatment.