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公开(公告)号：US20210338773A1
公开(公告)日：2021-11-04
申请号：US17282502
申请日：2018-10-17
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , SORBONNE UNIVERSITE , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE - CNRS -
摘要： The present invention relates to a new method for treating a patient suffering from a retinal degenerative disease. The Inventors discovered that nucleolin (NCL) is responsible in rods of the production of the short messenger of NXNL1 gene encoding RdCVF, a crucial factor for cones survival. Thus, the administration of NCL into the retina or the overexpression of NCL in recombinant cones to be transplanted into the retina, leads to a RdCVF expression and secretion by the cones themselves in order to encourage their own survival in an autocrine manner through the BSG1/GLUT1 complex. Thus, the invention concerns nucleolin polynucleotide or polypeptide for use in the treatment of a retinal degenerative disease in a patient in need. The invention also relates to recombinant cone overexpressing NCL for use in the treatment of a retinal degenerative disease.
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公开(公告)号：US20190328846A1
公开(公告)日：2019-10-31
申请号：US16463245
申请日：2017-11-30
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) , SORBONNE UNIVERSITE
摘要： The present invention relates to methods and pharmaceutical compositions for the treatment of retinal degenerative diseases. The inventors identified a new key actor of the mechanism underlying the protective role of RdCVF: 6-phosphofructo-2-kinase/fructose-2,6-bisphosphatase 2 (PFKFB2). The inventors showed that PFKFB2 is expressed by cones in a rod-dependant manner. In particular, they showed that its expression follows the viability of cones: its expression is lost in an animal model retinitis pigmentosa. The inventors accumulated evidences that PFKFB2, especially its kinase domain, is involved in the mechanism of action of RdCVF. More particularly they showed that transduction of a polynucleotide encoding for PFKFB2 increases cone survival. In particular, the present invention relates to a method of treating a retinal degenerative disease in a subject in need thereof comprising administering to the subject a therapeutically effective amount of a polynucleotide encoding for the PFKFB2 kinase domain.
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公开(公告)号：US20180153962A1
公开(公告)日：2018-06-07
申请号：US15576027
申请日：2016-05-20
申请人： INSERM (Institut National de la Santé et de la Recherche Médicale) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE-CNRS , UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6) , THE REGENTS OF THE UNIVERSITY OF CALIFORNIA , THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
发明人： Thierry LEVEILLARD , John FLANNERY , Mei XIN , Leah BYRNE , José-Alain SAHEL , Emmanuelle CLERIN-LACHAPELLE , Sun JUNWEI , Jean BENNETT , Jeannette BENNICELLI
摘要： The present invention relates to the synergistic combination of the short and long Rod-Derived Cone Viability Factors and to methods for treating retinal degenerative diseases.
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公开(公告)号：US20230033235A1
公开(公告)日：2023-02-02
申请号：US17791625
申请日：2021-01-08
申请人： INSERM (INSTITUT NATIONAL DE LAS SANTÉ ET DE LA RECHERCHE MÉDICALE , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) , SORBONNE UNIVERSITÉ
发明人： Thierry LEVEILLARD , Xin MEI , Mariangela CORSI
IPC分类号： G01N33/68
摘要： The NXNL2 gene encodes by alternative splicing for a trophic factor RdCVF2 that enhances the function and the survival of neurons involved in long term memory. Now the inventors demonstrated that the cell surface receptor for the trophic factor RdCVF2 is NPTN65. The set-up of methods that could be used to screen for small molecules, agonists of RdCVF2 signaling in the brain would be suitable for the development of a future metabolic and redox treatment of tauopathies and in particular Alzheimer's disease.
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公开(公告)号：US20210308169A1
公开(公告)日：2021-10-07
申请号：US15865354
申请日：2018-01-09
申请人： INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , Universite Pierre et Marie Curie (Paris 6) , Centre National de la Recherche Scientifique (CNRS)
发明人： Jose-Alain SAHEL , Serge PICAUD , Thierry LEVEILLARD , Deniz DALKARA , Jens DUEBEL , Botond ROSKA
IPC分类号： A61K31/7088 , A61K38/16 , C07K14/47 , C07K14/215 , A61K38/17
摘要： The present invention relates to an isolated nucleic acid molecule comprising i) a nucleotide sequence coding for a hyperpolarizing light-gated ion channel or pump gene from an archeon or for a light-active fragment of said gene, or the nucleotide sequence and ii) a nucleotide sequence coding for a neurotrophic factor for use in the treatment of a retinal degenerative disease.
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公开(公告)号：US20160213701A1
公开(公告)日：2016-07-28
申请号：US15092129
申请日：2016-04-06
申请人： INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , Universite Pierre et Marie Curie (Paris 6) , Centre National de la Recherche Scientifique (CNRS)
发明人： Jose-Alain SAHEL , Serge PICAUD , Thierry LEVEILLARD , Deniz DALKARA , Jens DUEBEL , Botond ROSKA
IPC分类号： A61K31/7088
CPC分类号： A61K31/7088 , A61K38/16 , A61K38/164 , A61K38/1709 , A61K48/00 , C07K14/215 , C07K14/47 , Y02A50/465 , A61K2300/00
摘要： The present invention relates to an isolated nucleic acid molecule comprising i) a nucleotide sequence coding for a hyperpolarizing light-gated ion channel or pump gene from an archeon or for a light-active fragment of said gene, or the nucleotide sequence and ii) a nucleotide sequence coding for a neurotrophic factor for use in the treatment of a retinal degenerative disease.
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公开(公告)号：US20200318138A1
公开(公告)日：2020-10-08
申请号：US16956894
申请日：2018-12-21
申请人： SPARINGVISION , INSERM (Institut National de la Santé et Recherche Médicale) , SORBONNE UNIVERSITE
发明人： Thierry LEVEILLARD , Najate AÏT-ALI MAAMRI , Fréderic BLOND , José-Alain SAHEL , Géraldine PUEL , Emmanuelle CLERIN
IPC分类号： C12N15/86 , C07K14/435
摘要： The present invention relates to improved constructs comprising the short and long Rod-Derived Cone Viability Factors and to methods for treating retinal degenerative diseases.
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