PROBio LatAm – List of hosts accepting graduate students and postdocs

Name: Andreotti, Giuseppina  (She / Her)

Research Area(s): Molecular Bases of Disease

Affiliation: National Research Council-Institute of Biomolecular Chemistry

Country: Italy

Website 

Scientific Interests:

Stabilization of pathological mutants with low molecular weight drugs is a transversal approach that has been developed for different diseases. It can be applied when the instability of a protein is the first cause of pathology (it means that in these cases the mutations do not affect the active site). Low molecular weight drugs can act by interacting with the affected protein directly (in this case they are defined pharmacological chaperones PC, PCs bind to and stabilize the specific target protein) or stabilizing mutants indirectly (in this case they are defined small molecules and they can act as proteasome inhibitors or co-inducers of HSPs, osmolytes etc). PCs and small molecules could act in synergy. In Fabry disease some mutations destabilize lysosomal alpha-galactosidase and cause the accumulation of Gb3. A pharmacological chaperone is already available but, regrettably, it is an inhibitor of the enzyme. The therapy is limited to a group of patients and needs a precise dosage and regimen to balance the stabilizing effect of the drug, which is required, versus the inhibitory effect, which is detrimental.
The therapeutic approach with small molecules can be extended to diseases without a cure such as PMM2-CDG. In this case all patients retain residual enzymatic activity and should be eligible for the therapy. The ultimate goal in both cases are drugs that stabilize pathogenic instable mutants. In addition to drug repositioning, that should be considered as the first option in the development of pharmacological therapies for rare diseases, GA is also interested in the search for new molecules among the natural products.

This offer applies to trainees/researchers eligible for the following fellowships

PROBio-Africa Fellowships, PROBio-LatAm Fellowships

Contact:

giuseppina.andreotti@cnr.it