Gene editing as the future of cardiac amyloidosis therapeutics
Cardiac Amyloidosis (CA) is a manifestation of a systemic disorder resulting from the
deposition of transthyretin (TTR) in the myocardium. This leads to a myriad of manifestations
ranging from conduction defects to heart failure. Previously CA was considered a rare
disease, but recent advances in diagnostics and therapeutics have revealed the prevalence
to be higher than estimated. There are two major classes of treatments for TTR cardiac
amyloidosis (ATTR-CA): TTR stabilizers, such as tafamidis and AG10, and RNA interference …
deposition of transthyretin (TTR) in the myocardium. This leads to a myriad of manifestations
ranging from conduction defects to heart failure. Previously CA was considered a rare
disease, but recent advances in diagnostics and therapeutics have revealed the prevalence
to be higher than estimated. There are two major classes of treatments for TTR cardiac
amyloidosis (ATTR-CA): TTR stabilizers, such as tafamidis and AG10, and RNA interference …
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