Updates to our CMT2A Research Project


In the Laboratory of Neural Stem Cells of the “Centro Dino Ferrari” of the University of Milan – Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Prof. Stefania Corti and her team of expert researchers work day after day on the project which is aiming at the development of a possible effective gene therapy for Charcot-Marie-Tooth type 2A (CMT2A).

Among the possible therapeutic approaches, “gene therapy aiming at correcting the underlying cause of the disease itself represents a rational therapeutic approach” for our Scientific Committee, as a type of strategy that has already given promising results in clinical trials for other neurodegenerative diseases. “Thus, by leveraging the growing knowledge in this area, we set out to use the same approach for the CMT2A” our researchers say. However, in the case of this pathology, given its peculiarities, “the development of a gene therapy requires additional efforts that result in a greater number of experiments and therefore in longer times”.

We must therefore be patient! Our researchers are optimistic and believe that the results achieved in these years of research represent the “necessary steps to arrive at an effective clinical trial in patients in the shortest possible time”. Of course, we will keep you updated, as we always have. Our Scientific Committee, in fact, keeps us constantly informed about the developments of research, advances that are regularly published on our website in the section Disease & Research.

We recently received and published:

1) the updating of our research project
2) integration with the transgenic mouse models
3) the table of the various types of CMT
4) a list of mutations to be added to our database

We thank with great pride and gratitude all the members of our Scientific Committee for the valuable work they are carrying out and for the timely updates regarding the progress of the scientific research conducted.

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