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公开(公告)号:US20240360396A1
公开(公告)日:2024-10-31
申请号:US18580848
申请日:2022-07-21
Applicant: CENTRE D'ETUDE DES CELLULES SOUCHES (CECS) , UNIVERSITE D'EVRY VAL D'ESSONNE , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , UNIVERSITÉ PARIS-SACLAY
Inventor: Christelle MONVILLE , Elise HERARDOT , Karim BEN M'BAREK , Frédéric HAMOUDA
CPC classification number: C12M25/02 , A61L27/3813
Abstract: The preparation of a microstructured scaffold corresponding to a biocompatible membrane-type support material for cell culture, and a composition that includes the microstructured scaffold and cells, in particular photoreceptors. Also, the preparation of an implantable graft for the treatment of diseases of the retinal pigment epithelium and the neural retina.
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公开(公告)号:US20240209030A1
公开(公告)日:2024-06-27
申请号:US18024103
申请日:2021-09-10
Applicant: Genethon , Universite d'Evry Val d'Essonne , Institut National de la Santé et de la Recherche Médicale
Inventor: Isabelle Richard , Natalia Dominguez
IPC: C07K14/005 , C12N15/86
CPC classification number: C07K14/005 , C12N15/86 , C12N2750/14122 , C12N2750/14143
Abstract: The invention relates to a peptide-modified AAV capsid with improved tropism, in particular increased muscle tropism and/or reduced liver tropism. The invention relates also to the derived recombinant AAV vector particle packaging a gene of interest, and its use in gene therapy, in particular for treating muscle diseases.
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公开(公告)号:US20230338369A1
公开(公告)日:2023-10-26
申请号:US17789128
申请日:2020-12-26
Applicant: Centre d'Etude des Cellules Souches (CECS) , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE , Genethon , UNIVERSITE D'EVRY-VAL-D'ESSONNE
Inventor: Lucile HOCH , Xavier NISSAN , Isabelle RICHARD
IPC: A61K31/4965 , A61K38/06 , A61K31/5355 , A61K31/167 , A61K31/18 , A61K31/422 , A61K45/06
CPC classification number: A61K31/4965 , A61K38/06 , A61K31/5355 , A61K31/167 , A61K31/18 , A61K31/422 , A61K45/06
Abstract: The present invention relates to the combination of a proteasome inhibitor and a histone deacetylase (HDAC) inhibitor and its use for the treatment of a genetic disease linked to a conformational disorder of at least one protein, said disorder causing the proteasome degradation of the protein.
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公开(公告)号:US20230313152A1
公开(公告)日:2023-10-05
申请号:US18022534
申请日:2021-08-23
Applicant: GENETHON , INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , UNIVERSITE D'EVRY VAL D'ESSONNE
Inventor: GIUSEPPE RONZITTI , PATRICE VIDAL
CPC classification number: C12N9/1051 , C12Y204/01025 , C12N15/86 , A61P3/00 , C12N2750/14143
Abstract: The present invention relates to a functional C-terminal truncated GDE polypeptide for the treatment of glycogen storage disease III.
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公开(公告)号:US11754561B2
公开(公告)日:2023-09-12
申请号:US15149833
申请日:2016-05-09
Applicant: GENOPTICS , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE , UNIVERSITE D'EVRY VAL D'ESSONNE
Inventor: Régis Daniel , Florence Gonnet , William Buchmann , Sophie Bellon , Nathalie Jarroux , Marielle Anger-Leroy
IPC: G01N33/543 , G01N21/552 , G01N33/553 , G01N33/68
CPC classification number: G01N33/54373 , G01N21/554 , G01N33/54353 , G01N33/553 , G01N33/6851 , G01N2560/00 , G01N2570/00 , Y10T436/24
Abstract: The invention relates to a method for coupling in-line the analysis of molecular interactions by surface plasmon resonance (SPR) with a structural identification by mass spectrometry using the same functionalized support for both types of analysis.
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公开(公告)号:US20230250450A1
公开(公告)日:2023-08-10
申请号:US18009025
申请日:2021-06-09
Applicant: Genethon , Universite d'Evry Val d'Essonne , INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE
Inventor: Isabelle Richard
CPC classification number: C12N15/86 , A61P9/10 , A61K38/1709 , C12N2750/14143 , C12N2750/14171 , A61K48/00
Abstract: The present invention relates to the treatment of dilated cardiomyopathies, in particular to the use of an activator of NRF2.
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公开(公告)号:US20220275396A1
公开(公告)日:2022-09-01
申请号:US17727990
申请日:2022-04-25
Applicant: GENETHON , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , UNIVERSITE D'EVRY VAL D'ESSONNE
Inventor: FEDERICO MINGOZZI , GIUSEPPE RONZITTI
IPC: C12N15/86 , A61K35/34 , A61K35/407
Abstract: The present invention relates to variants of acid-alpha glucosidase and uses thereof.
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公开(公告)号:US11384351B2
公开(公告)日:2022-07-12
申请号:US16633768
申请日:2018-07-27
Applicant: INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , UNIVERSITE D'EVRY—VAL D'ESSONNE
Inventor: David Pastre , Bénédicte Desforges , Alexandre Maucuer
IPC: C12Q1/68 , C12N15/10 , C12Q1/6806
Abstract: The present invention relates to the field of nucleic acid purification. In particular, it relates to methods and tools for purifying nucleic acids in a sample; which are compatible with high-throughput sequencing and diagnosis. The inventors have shown that nucleic acid binding proteins recruited to polymerized tubulin (i.e. microtubules) could, subsequently, be isolated from cell lysates. Surprisingly, it has now been found that the amount of recovered nucleic acid found in these microtubule pellets increases dramatically in the presence of nucleic acid-trapping proteins comprising a nucleic acid-binding moiety and a polymerized tubulin-binding moiety, by comparison to proteins devoid of the nucleic acid-binding moiety; and that the recovery of the purified nucleic acids was itself particularly efficient. This purification method is particularly amenable to high-throughput sequencing and/or in the context of a diagnosis method for identifying or comparing the amount of nucleic acids in a set of samples.
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公开(公告)号:US20220162640A1
公开(公告)日:2022-05-26
申请号:US17601934
申请日:2020-04-07
Applicant: GENETHON , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) , SORBONNE UNIVERSITE , UNIVERSITE D'EVRY VAL D'ESSONNE
Inventor: GIUSEPPE RONZITTI , PATRICE VIDAL , FEDERICO MINGOZZI
Abstract: The present invention relates to hybrid promoters to drive gene expression in muscles.
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公开(公告)号:US11298348B2
公开(公告)日:2022-04-12
申请号:US16960503
申请日:2019-01-10
Applicant: INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , UNIVERSITE D'EVRY-VAL-D'ESSONNE , GENETHON , UNIVERSITA 'DEGLI STUDI DI PADOVA
Inventor: Isabelle Richard , Dorianna Sandona
IPC: A61K31/47 , A61P21/00 , A61K31/427
Abstract: Sarcoglycanopathies are autosomal recessive diseases caused by mutations in the one of the genes coding for any sarcoglycans (SG). The inventors previously showed that the application of small molecules developed to rescue ΔF508-CFTR trafficking, and known as CFTR correctors, improved the maturation of several α-sarcoglycan mutants that were consequently rescued at the plasma membrane (WO 014086687). Now, the inventors show that some specific CFTR correctors provide additive and even synergic effect when administered in combination.
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