Therapeutic Potential of Polyamidoamine Dendrimer for Amyloidogenic Transthyretin Amyloidosis.

08:00 EDT 26th March 2019 | BioPortfolio

Summary of "Therapeutic Potential of Polyamidoamine Dendrimer for Amyloidogenic Transthyretin Amyloidosis."

Amyloidogenic transthyretin (ATTR) amyloidosis is caused by a formation of ATTR amyloid fibrils. Because ATTR misfolding triggers the formation of aggregates and amyloid fibrils, which are considered to deposit on the tissues, novel clinically effective therapeutic strategies targeted to those processes are urgently needed. In this study, to discover a new drug candidate for ATTR amyloidosis therapy, we focused on polyamidoamine dendrimer (dendrimer), a 3D-structural nanomaterial, which has a branched cationic polymer repeating polyamidoamine units. Dendrimer (G2) not only inhibited ATTR V30M amyloid fibril formation, but also reduced already formed ATTR V30 M amyloid fibrils by reducing β-sheet structure of ATTR V30 M protein. Moreover, intravenous administration of dendrimer (G2) reduced TTR deposition in human ATTR V30 M transgenic rats. These results indicate that dendrimer (G2) may possess both inhibitory and breaking effects on ATTR V30 M amyloid, suggesting that dendrimer has the potential as a dual effective agents against TTR amyloidosis.


Journal Details

This article was published in the following journal.

Name: ACS chemical neuroscience
ISSN: 1948-7193


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