Zobrazeno 1 - 10
of 39
pro vyhledávání: '"Hasane Ratni"'
Autor:
Hasane Ratni
Publikováno v:
CHIMIA, Vol 75, Iss 7/8, Pp 614-619 (2021)
Nervous system disorders affect millions of people around the world, through a very broad range of diseases. Here we describe our contribution to find a treatment for patients suffering from three of those diseases. The first one, autism spectrum dis
Externí odkaz:
https://doaj.org/article/e108d4dcd61f4c3892c0768531c2e9fd
Autor:
Manaswini Sivaramakrishnan, Kathleen D. McCarthy, Sébastien Campagne, Sylwia Huber, Sonja Meier, Angélique Augustin, Tobias Heckel, Hélène Meistermann, Melanie N. Hug, Pascale Birrer, Ahmed Moursy, Sarah Khawaja, Roland Schmucki, Nikos Berntenis, Nicolas Giroud, Sabrina Golling, Manuel Tzouros, Balazs Banfai, Gonzalo Duran-Pacheco, Jens Lamerz, Ying Hsiu Liu, Thomas Luebbers, Hasane Ratni, Martin Ebeling, Antoine Cléry, Sergey Paushkin, Adrian R. Krainer, Frédéric H.-T. Allain, Friedrich Metzger
Publikováno v:
Nature Communications, Vol 8, Iss 1, Pp 1-13 (2017)
Small molecules correcting the splicing deficit of the survival of motor neuron 2 (SMN2) gene have been identified as having therapeutic potential. Here, the authors provide evidence that SMN2 mRNA forms a ribonucleoprotein complex that can be specif
Externí odkaz:
https://doaj.org/article/86ed63c63e664f938352b802e813bfcc
Autor:
Aleksandra Wlodek, Steve G. Kendrew, Nigel J. Coates, Adam Hold, Joanna Pogwizd, Steven Rudder, Lesley S. Sheehan, Sarah J. Higginbotham, Anna E. Stanley-Smith, Tony Warneck, Mohammad Nur-E-Alam, Markus Radzom, Christine J. Martin, Lois Overvoorde, Markiyan Samborskyy, Silke Alt, Daniel Heine, Guy T. Carter, Edmund I. Graziani, Frank E. Koehn, Leonard McDonald, Alexander Alanine, Rosa María Rodríguez Sarmiento, Suzan Keen Chao, Hasane Ratni, Lucinda Steward, Isobel H. Norville, Mitali Sarkar-Tyson, Steven J. Moss, Peter F. Leadlay, Barrie Wilkinson, Matthew A. Gregory
Publikováno v:
Nature Communications, Vol 8, Iss 1, Pp 1-10 (2017)
Reengineering polyketide synthase encoding genes to produce analogues of natural products can be slow and low-yielding. Here the authors use accelerated evolution to recombine the gene cluster for rapid production of rapamycin-related products.
Externí odkaz:
https://doaj.org/article/b36ef21246ab47bfb8cbd4e969caea7c
Publikováno v:
The Handbook of Medicinal Chemistry ISBN: 9781788018982
RNA as a drug target offers a wealth of opportunities to therapeutically modulate numerous cellular processes, including those linked to the so-called “undruggable” protein targets. Of particular interest is the modulation of the natural process
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::c5a873f6591c39f7929a54ddf6f4fe35
https://doi.org/10.1039/9781788018982-00830
https://doi.org/10.1039/9781788018982-00830
Development and Optimization of the Manufacturing Process for RNA-Splicing Modifier Risdiplam RG7916
Autor:
Christian Moessner, Fabienne Hoffmann-Emery, Jean-Michel Adam, Serena Fantasia, Dan Fishlock, Roland Meier, Georg Wuitschik, Hasane Ratni
Publikováno v:
ACS Symposium Series ISBN: 9780841297333
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::57dab2446985237000eeb07648e81769
https://doi.org/10.1021/bk-2022-1423.ch008
https://doi.org/10.1021/bk-2022-1423.ch008
Autor:
Nicole Risher, Josephine Sheedy, Hasane Ratni, Zhihua Feng, Xin Zhao, Amal Dakka, Anna Mollin, Catherine Lutz, Karen K. Y. Ling, Ellen Welch, Friedrich Metzger, Marla Weetall, Karen S. Chen, Jana Narasimhan, Nikolai Naryshkin, John D. Baird, Chien-Ping Ko
Publikováno v:
Human Molecular Genetics. 31:82-96
Spinal muscular atrophy (SMA) is caused by the loss of the survival motor neuron 1 (SMN1) gene function. The related SMN2 gene partially compensates but produces insufficient levels of SMN protein due to alternative splicing of exon 7. Evrysdi™ (ri
Autor:
Michael Reutlinger, Georg Schmitt, Roland Jakob-Roetne, Anja Limberg, Hasane Ratni, Werner Neidhart, Solen Pichereau, Caterina Bissantz, Karlheinz Baumann, Rosa-Maria Rodriguez-Sarmiento, Mingqiu Lu, Bjoern Bartels, Irene Gerlach, André Alker, Eric Zhang, Chen Weichun
Publikováno v:
ACS Med Chem Lett
[Image: see text] γ-Secretase (GS) is a key target for the potential treatment of Alzheimer’s disease. While inhibiting GS led to serious side effects, its modulation holds a lot of potential to deliver a safe treatment. Herein, we report the disc
Autor:
A F Stepan, Michael Reutlinger, X A Cook, Karlheinz Baumann, Peter Bellotti, Luke Green, Walter Vifian, Thomas Luebbers, Hasane Ratni
Publikováno v:
RSC Med Chem
Phenyl rings are one of the most prevalent structural moieties in active pharmaceutical ingredients, even if they often contribute to poor physico-chemical properties. Herein, we propose the use of a bridged piperidine (BP) moiety as a phenyl bioisos
Publikováno v:
ACS Med Chem Lett
[Image: see text] Not too long ago, the concept of selectively targeting mRNA with small molecules was perceived as a formidable scientific challenge. The discovery of small molecule splicing modifiers and the development of risdiplam for the treatme
Autor:
Schnider Patrick, Erwin Goetschi, Neil Parrott, Hasane Ratni, Markus von Kienlin, Basil Künnecke, Celine Risterucci, Cosimo Dolente, Mark Rogers-Evans, Roland Jakob-Roetne, Christophe Grundschober, Andreas Bruns, Caterina Bissantz, Emmanuel Pinard, Wolfgang Muster, Thomas Mueggler
Publikováno v:
Journal of medicinal chemistry. 63(4)
We recently reported the discovery of a potent, selective, and brain-penetrant V1a receptor antagonist, which was not suitable for full development. Nevertheless, this compound was found to improve surrogates of social behavior in adults with autism