Rescue of cardiomyopathy through U7snRNA‐mediated exon skipping in Mybpc3‐targeted knock‐in mice

Autor: Christina Gedicke‐Hornung, Verena Behrens‐Gawlik, Silke Reischmann, Birgit Geertz, Doreen Stimpel, Florian Weinberger, Saskia Schlossarek, Guillaume Précigout, Ingke Braren, Thomas Eschenhagen, Giulia Mearini, Stéphanie Lorain, Thomas Voit, Patrick A. Dreyfus, Luis Garcia, Lucie Carrier

Publikováno v: EMBO Molecular Medicine, Vol 5, Iss 7, Pp 1128-1145 (2013)

Abstract Exon skipping mediated by antisense oligoribonucleotides (AON) is a promising therapeutic approach for genetic disorders, but has not yet been evaluated for cardiac diseases. We investigated the feasibility and efficacy of viral‐mediated A

Externí odkaz: https://doaj.org/article/2151f46ca23e4c23b5b19581f81c7297

Influence of Fumed Silicon Dioxide on the Stabilization of Eudragit® RS/RL 30 D Film-Coated Theophylline Pellets

Autor: A. Waseem Malick, Navnit Hargovindas Shah, Shawn A. Kucera, Doreen Stimpel, James W. McGinity, Martin Howard Infeld

Publikováno v: Pharmaceutical Development and Technology. 13:245-253

The objective of this study was to investigate the influence of various grades of fumed silicon dioxide on the drug release rate and physical aging of theophylline pellets coated with Eudragit RS 30 D and RL 30 D. Free films were assessed for both ph

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c00bf9a163d3bb9a3e89c2752ad1823d
https://doi.org/10.1080/10837450801949665

Repair of Mybpc3 mRNA by 5′-trans-splicing in a Mouse Model of Hypertrophic Cardiomyopathy

Autor: Luis Garcia, Hugo A. Katus, Ingke Braren, Christina Gedicke-Hornung, Elisabeth Krämer, Doreen Stimpel, Giulia Mearini, Guillaume Précigout, Stéphanie Lorain, Oliver J. Müller, Thomas Voit, Birgit Geertz, Thomas Eschenhagen, Lucie Carrier

Publikováno v: Molecular Therapy-Nucleic Acids
Molecular Therapy-Nucleic Acids, Elsevier, 2013, 2, pp.e102. ⟨10.1038/mtna.2013.31⟩
Molecular Therapy: Nucleic Acids, Vol 2, Iss C (2013)
Molecular Therapy—Nucleic Acids
Molecular Therapy-Nucleic Acids, 2013, 2, pp.e102. ⟨10.1038/mtna.2013.31⟩
Molecular Therapy. Nucleic Acids

RNA trans-splicing has been explored as a therapeutic option for a variety of genetic diseases, but not for cardiac genetic disease. Hypertrophic cardiomyopathy (HCM) is an autosomal-dominant disease, characterized by left ventricular hypertrophy (LV

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bce2215baf13c470a505a969f9150e9c