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 - Summary
Dec 16, 2016
Angewandte Chemie International Edition
Miller JB, Zhang S, Kos P, Xiong H, Zhou K, Perelman SS, Zhu H, Siegwart DJ
The CRISPR/Cas (clustered regularly interspaced short palindromic repeat/CRISPR-associated protein) gene-editing tool allows for sequence-specific cuts to genomic DNA, which can result in permanent silencing of a gene, or replacement of gene sequen...
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