Saturday, 18th November 2017

 Molecular Neuropathology

    Alternative therapies for metabolic diseases

 


Grupo-400

 


 

 

Alejandra Gámez Abascal

DSciStaff

DPublications

 

 

 

 

Research summary:

 

The aim of this project is the development of alternative therapies for inherited metabolic diseases for which there is no treatment available and in which the prognosis is most often fatal.To reach this goal the methodological strategies can be encompassed in two complementary therapeutic approaches:

- Enzyme therapy in order to eliminate the toxic metabolites produced as a result of an enzymatic defect using chemical modified proteins capable of metabolize the excess of these toxic compounds.

- Enzyme therapy in order to eliminate the toxic metabolites produced as a result of an enzymatic defect using chemical modified proteins capable of metabolize the excess of these toxic compounds.

The global goal is to screen and select different enzyme candidates and pharmacological chaperones that can be potential therapeutic targets based on their biological function to diminish the excess accumulation of toxic metabolites or increase the catalytic activity of the altered proteins respectively.