Add to ORKGFrameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated cardiomyopathy (DCM), a heart disease characterized by ventricular dilatation, systolic dysfunc-tion, and progressive heart failure. To date, there are no specific treatment options for DCM patients but heart transplantation. Here, we show the beneficial potential of reframing titin tran-scripts by antisense oligonucleotide (AON)-mediated exon skipping in human and murine models of DCM carrying a previously identi-fied autosomal-dominant frameshift mutation in titin exon 326. Correction of TTN reading frame in patient-specific cardiomyocytes derived from induced pluripotent stem cells rescued defective myofibril assembly and stability and no...
Thesis (Ph.D.)--University of Washington, 2018The giant sarcomere protein titin plays a number of im...
Thesis (Ph.D.)--University of Washington, 2018The giant sarcomere protein titin plays a number of im...
The recent discovery of heterozygous human mutations that truncate full-length titin (TTN, an abunda...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Non-ischemic dilated cardiomyopathy (DCM) is one of the most frequent pathologies requiring cardiac ...
BACKGROUND: Titin truncation variants (TTNtvs) are the most common inheritable risk factor for dilat...
<p><b><i>Objectives:</i></b> Dilated cardiomyopathy (DCM) is a leading cause of sudden cardiac death...
5siDilated cardiomyopathy (DCM) is a leading cause of heart failure, sudden cardiac death and heart ...
Thesis (Ph.D.)--University of Washington, 2018The giant sarcomere protein titin plays a number of im...
Thesis (Ph.D.)--University of Washington, 2018The giant sarcomere protein titin plays a number of im...
The recent discovery of heterozygous human mutations that truncate full-length titin (TTN, an abunda...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated...
Non-ischemic dilated cardiomyopathy (DCM) is one of the most frequent pathologies requiring cardiac ...
BACKGROUND: Titin truncation variants (TTNtvs) are the most common inheritable risk factor for dilat...
<p><b><i>Objectives:</i></b> Dilated cardiomyopathy (DCM) is a leading cause of sudden cardiac death...
5siDilated cardiomyopathy (DCM) is a leading cause of heart failure, sudden cardiac death and heart ...
Thesis (Ph.D.)--University of Washington, 2018The giant sarcomere protein titin plays a number of im...
Thesis (Ph.D.)--University of Washington, 2018The giant sarcomere protein titin plays a number of im...
The recent discovery of heterozygous human mutations that truncate full-length titin (TTN, an abunda...