Information about ATTR Amyloidosis | Alnylam
Information about ATTR Amyloidosis | Alnylam
Apr 4, 2022
alnylam.com
Amyloidosis is a rare disease that occurs when abnormal proteins build up in tissues and organs, affecting their function. Dr. Amy Kontorovich breaks down the different types of amyloidosis, the early warning signs to watch for, and who may be most at risk. Dr. Izzy Kornblau explains what a diagnosis means for family members. Understanding these key factors can lead to earlier detection and better outcomes. #AmyloidosisAwareness | The Mount Sinai Hospital
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Amyloidosis is a rare disease that occurs when abnormal proteins b…
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Cardiac amyloidosis — Treatment options
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Daily CRISPR Updates on Instagram: "Researchers recently investigated the use of SpCas9-based gene editing to target the TTR gene associated with ATTR amyloidosis, a severe condition caused by misfolded TTR proteins. Their engineered SpCas9-Mut5 demonstrates significantly improved fidelity with minimal off-target edits. SpCas9-Mut5, tested in human cell lines and animal models, effectively lowers TTR protein levels, offering long-term reduction with sustained effects. Additionally, it improves t
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