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10.15252/emmm.201505047 http://scihub22266oqcxt.onion/10.15252/emmm.201505047 C4492817!4492817!25759365     free     free     free Deprecated: Implicit conversion from float 231.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 231.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 231.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 231.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 231.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       EMBO+Mol+Med 2015 ; 7 (5): 562-76 Nephropedia Template TP {{tp|p=25759365|t=2015. Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy |pdf=|usr=}}{{25759365}} gab.com Text Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy JO: EMBO Mol Med http://www.kidney.de/pget.php?&tw=1&pmid=25759365 #FOAvip Frameshift 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 dysfunction, 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 transcripts by antisense oligonucleotide (AON)-mediated exon skipping in human and murine models of DCM carrying a previously identified 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 normalized the sarcomeric protein expression. AON treatment in Ttn knock-in mice improved sarcomere formation and contractile performance in homozygous embryos and prevented the development of the DCM phenotype in heterozygous animals. These results demonstrate that disruption of the titin reading frame due to a truncating DCM mutation can be restored by exon skipping in both patient cardiomyocytes in vitro and mouse heart in vivo, indicating RNA-based strategies as a potential treatment option for DCM. Twit Text FOAVip Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy ????EMBO Mol Med http://www.kidney.de/pget.php?&tw=1&pmid=25759365 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=25759365 #FOAvip English Wikipedia <ref>{{Cite pmid|25759365}}</ref> Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy #MMPMID25759365 Gramlich M; Pane LS; Zhou Q; Chen Z; Murgia M; Schötterl S; Goedel A; Metzger K; Brade T; Parrotta E; Schaller M; Gerull B; Thierfelder L; Aartsma-Rus A; Labeit S; Atherton JJ; McGaughran J; Harvey RP; Sinnecker D; Mann M; Laugwitz KL; Gawaz MP; Moretti A EMBO Mol Med 2015[May]; 7 (5): 562-76 PMID25759365show ga Frameshift 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 dysfunction, 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 transcripts by antisense oligonucleotide (AON)-mediated exon skipping in human and murine models of DCM carrying a previously identified 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 normalized the sarcomeric protein expression. AON treatment in Ttn knock-in mice improved sarcomere formation and contractile performance in homozygous embryos and prevented the development of the DCM phenotype in heterozygous animals. These results demonstrate that disruption of the titin reading frame due to a truncating DCM mutation can be restored by exon skipping in both patient cardiomyocytes in vitro and mouse heart in vivo, indicating RNA-based strategies as a potential treatment option for DCM. äAntisense-mediated exon skipping: a therapeutic strategy for titin-bas(epmc).pdf DeepDyve Pubget Overpricing