Abstract
Transthyretin (TTR) amyloidosis is a protein misfolding disorder where tetramers of TTR dissociate, causing the formation of insoluble amyloids leading to organ dysfunction and damage. Worldwide, the disease currently affects between 5,000-10,000 people and many novel therapeutic techniques are appearing with the potential to treat TTR amyloidosis. Of the non-CRISPR treatments, they can be broken down into three categories: TTR stabilizers, human monoclonal antibodies, and gene silencers. In this paper, I will discuss both the current state of research and treatment of TTR amyloidosis treatments, as well as the role of CRISPR’s potential future therapeutic applications.
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