Scientific publications

Synthesis of Stable Cholesteryl−Polyethylene Glycol−Peptide Conjugates with Non-Disperse Polyethylene Glycol Lengths, published in ACS Omega 2020, 5, 5508−5519

A method for conjugating cholesterol to peptide ligands through non-disperse polyethylene glycol (ND-PEG) through a non-hydrolysable linkage is described. The iterative addition of tetraethylene glycol macrocyclic sulfate to cholesterol (Chol) renders a family of highly pure well-defined Chol-PEG compounds with different PEG lengths from 4 up to 20 ethylene oxide units, stably linked through an ether bond. The conjugation of these Chol-PEG compounds to the cyclic (RGDfK) peptide though Lys5 side chains generates different lengths of Chol-PEG-RGD conjugates that retain the oligomer purity of the precursors, as analysis by HRMS and NMR has shown. Other derivatives were synthesized with similar results, such as Chol-PEG-OCH3 and Chol-PEG conjugated to glutathione and Tf1 peptides through maleimide−thiol chemoselective ligation. This method allows the systematic synthesis of highly pure uniform stable Chol-PEGs, circumventing the use of activation groups on each elongation step and thus reducing the number of synthesis steps.

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Hospital Vall D´Hebron in collaboration with the H2020 funded Smart 4 Fabry project celebrated a seminar on Lisosomal Disorders, with a special focus on Fabry Disease.

Researchers and clinicians working in the field of lysosomal disorders had the opportunity to put in common the most recent advances in the diagnosis, pathophysiology and management of lysosomal storage disorders in general and of Fabry Disease in particular. During the seminar celebrated in Barcelona on November 19th, patients also had the chance to discuss with administrators and professionals about their expectations and demands, as well as their the need for collaborative actions aiming at designing plans for early diagnosis and easy access to the most suitable therapies.