Publication - Summary
Feb 27, 2018
Cell Reports
J. Finn, A. Smith, M. Patel, L. Shaw, M. Youniss, J. Heteren, T. Dirstine, C. Ciullo, R. Lescarbeau, J. Seitzer, R. Shah, A. Shah, D. Ling, J. Growe, M. Pink, E. Rohde, K. Wood, W. Salomon, W. Harrington, C. Dombrowski, W. Strapps, Y. Chang, D. Morrissey
Hereditary transthryretin amyloidosis is a rare disease caused by mutations in the gene encoding the protein transthyretin (TTR), causing it to misfold into amyloid plaques, leading to debilitating symptoms. In 2018,...
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Publication - Abstract
Feb 20, 2020
Nature Communications
S. Patel, N. Ashwanikumar, E. Robinson, Y. Xia, C. Mihai, J.P. Griffith III, S. Hou, A.A. Esposito, T. Ketova, K. Welsher, J.L Joyal, Ö. Almarsson and G. Sahay
Endosomal sequestration of lipid-based nanoparticles (LNPs) remains a formidable barrier to delivery. Herein, structure-activity analysis of cholesterol analogues reveals that incorporation of C-24 alkyl phytosterols into LNPs (eLNPs) enhances gene transfection and the length of ...
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