Advance in the gene editing in the treatment of transthyretin cardiac amyloidosis
XIE Lin-feng
LUO Su-xin
HUANG Bi
Abstract：Transthyretin cardiac amyloidosis(ATTR-CM)is caused by the deposition of insoluble transthyroxine(TTR)protein in the myocardium,leading to various complications including cardiac conduction abnormalities and heart failure.In the past,the treatment of ATTR-CM only focused on its complications such as heart failure and arrhythmia,lacking effective drugs for etiology.In recent years,with the emergence of stabilizers for TTR such as tafamidis acid and RNA interference(SiRNA)drugs such as patisiran,the treatment of ATTR-CM has truly entered the era of etiological treatment.Gene editing is a new method that differs from traditional drug therapy mechanisms.It uses CRISPR/Cas9 to edit target genes and achieves the change from protein to clinical phenotype.It has been confirmed in the animal model of ATTR-CM that CRISPR/Cas9 gene editing can effectively reduce the deposition of TTR protein in tissues.Clinical studies on small samples have also achieved similar results as animal experiments,which may have a revolutionary impact on the treatment of ATTR-CM in the future.This article provides a review of the current progress in gene editing technology for the treatment of ATTR-CM.
Keywords：Transthyretin cardiac amyloidosisHeart failureArrhythmiaGene editing
Publication Date：2024-03-10
Online Publishing Date：2025-08-15(First online date of this platform, not the publication date of the document)
Pages：5( 237-241 )