NEW YORK (GenomeWeb) – Arcturus Therapeutics has announced that it has added a second drug candidate to its transthyretin-mediated amyloidosis (ATTR) program.

ATTR disease is caused by mutations in the transthyretin (TTR) gene, which triggers the accumulation of abnormal amyloid proteins in the body. Previously, Arcturus announced that it is developing an ATTR drug designed to silence both the wild-type and mutant forms of the gene, which would be used for patients with a form of ATTR that primarily damages heart tissue called familial amyloidotic cardiomyopathy (FAC).

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Sep
12
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This webinar will cover recent advances in the use of CRISPR for generating animal models and cell lines.