Patent
Treatment of ciliopathies
Carsten Rudolph,Verena Kretzschmann,Rebekka Kubisch-Dohmen,Christian Dohmen,Johannes Geiger,Aneja Manish Kumar,Weiss Ludwig,Heymut Omran,Petra Pennekamp,Kai Wohlgemuth,Sandra Cindric,Niki Tomas Loges,Johanna Raidt,Adrian Ter Steege +13 more
- 20 Aug 2020
TL;DR: In this paper, a pharmaceutical composition comprising a polyribonucleotide for use in treating a ciliopathy in a subject suffering of a Ciliopathy, wherein the polyribone encodes a functional version of a protein a defect of which is associated with said ciliopathies, and wherein administration of said pharmaceutical composition to the respiratory system of said subject is effected when the subject shows an inflammation of the respiratory systems.
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Abstract: The present disclosure provides a pharmaceutical composition comprising a polyribonucleotide for use in treating a ciliopathy in a subject suffering of a ciliopathy, wherein the polyribonucleotide encodes a functional version of a protein a defect of which is associated with said ciliopathy, and wherein administration of said pharmaceutical composition to the respiratory system of said subject is effected when the subject shows an inflammation of the respiratory system. Further, the present disclosure relates to a method for analyzing the effect of a polyribonucleotide on ciliogenesis, wherein said polyribonucleotide encodes a protein involved in and/or required for ciliogenesis.
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References
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Molecular Cell Biology
Harvey F. Lodish
- 01 Jan 1986
TL;DR: Molecular cell biology, Molecular cell biology , مرکز فناوری اطلاعات و اصاع رسانی, کδاوρزی
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Functional genomic screen for modulators of ciliogenesis and cilium length
Joon Kim,Ji Eun Lee,Susanne Heynen-Genel,Eigo Suyama,Keiichiro Ono,Ki-Young Lee,Ki-Young Lee,Trey Ideker,Pedro Aza-Blanc,Joseph G. Gleeson +9 more
TL;DR: A critical regulatory step that couples actin dynamics and endocytic recycling with ciliogenesis is uncovered, and also provides potential target molecules for future study.
Patent
Lipid encapsulated interfering RNA
Ian MacLachlan,Ellen Grace Ambegia,James Heyes +2 more
- 07 Jun 2005
TL;DR: In this article, the authors provide compositions and methods for silencing gene expression by delivering nucleic acid-lipid particles comprising a siRNA molecule to a cell, where the siRNA is used to identify the gene expression.
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An siRNA-based functional genomics screen for the identification of regulators of ciliogenesis and ciliopathy genes
Gabrielle Wheway,Miriam Schmidts,Dorus A. Mans,Katarzyna Szymanska,Thanh Minh T. Nguyen,Hilary E. Racher,Ian G. Phelps,Grischa Toedt,Julie Kennedy,Kirsten A. Wunderlich,Nasrin Sorusch,Zakia Abdelhamed,Subaashini Natarajan,Warren Herridge,Jeroen van Reeuwijk,Nicola Horn,Karsten Boldt,David A. Parry,Stef J.F. Letteboer,Susanne Roosing,Matthew Adams,Sandra M. Bell,Jacquelyn Bond,Julie Higgins,Ewan E. Morrison,Darren C. Tomlinson,Gisela G. Slaats,Teunis J. P. van Dam,Lijia Huang,Kristin Kessler,Andreas Giessl,Clare V. Logan,Evan A. Boyle,Jay Shendure,Shamsa Anazi,Mohammed A. Aldahmesh,Selwa A. Al Hazzaa,Selwa A. Al Hazzaa,Robert A. Hegele,Carole Ober,Patrick Frosk,Aizeddin A. Mhanni,Bernard N. Chodirker,Albert E. Chudley,Ryan E. Lamont,Francois P. Bernier,Chandree L. Beaulieu,Paul M. K. Gordon,Richard T. Pon,Clem Donahue,A. James Barkovich,Louis Wolf,Carmel Toomes,Christian Thiel,Kym M. Boycott,Martin McKibbin,Chris F. Inglehearn,Fiona Stewart,Heymut Omran,Martijn A. Huynen,Panagiotis I. Sergouniotis,Panagiotis I. Sergouniotis,Fowzan S. Alkuraya,Jillian S. Parboosingh,A. Micheil Innes,Colin E. Willoughby,Rachel H. Giles,Andrew R. Webster,Andrew R. Webster,Marius Ueffing,Marius Ueffing,Oliver E. Blacque,Joseph G. Gleeson,Uwe Wolfrum,Philip L. Beales,Toby J. Gibson,Dan Doherty,Hannah M. Mitchison,Ronald Roepman,Colin A. Johnson +79 more
TL;DR: A whole-genome siRNA-based reverse genetics screen for defects in biogenesis and/or maintenance of the primary cilium is described and insights into ciliogenesis complexity and roles for unanticipated pathways in human genetic disease are provided.
Patent
Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
Jon A. Wolff,James E. Hagstrom,Vladimir G. Budker,Jeffery D. Fritz +3 more
- 17 Jan 1998
TL;DR: In this paper, a process of transfecting a cell with a polynucleotide mixed with one or more amphipathic compounds and an effective amount of a DNA-binding protein is described.
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