Cardiac amyloidosis 101: Why early diagnosis and targeted treatment matter - Mayo Clinic News Network
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🧬ATTR amyloidosis! New therapies are emerging with diverse mechanisms such as protein stabilization, gene editing, mRNA silencing, and fibril clearance. 1. Tetramer stabilizers (tafamidis/acoramidis) that prevent amyloid formation. 2. CRISPR-Cas9 knockout techniques aiming to delete the problematic gene. 3. siRNA/ASO silencers (patisiran/vutrisiran) that reduce TTR protein production. 4. Monoclonal antibodies (ALXN2220) to clear amyloid deposits. #TTRAmyloidosis #InnovativeTherapies #MedicalRes
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