-
公开(公告)号：US20220380807A1
公开(公告)日：2022-12-01
申请号：US17861676
申请日：2022-07-11
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , UNIVERSITÉ D'EVRY-VAL-D'ESSONNE , GENETHON , FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA , FONDAZIONE TELETHON
IPC分类号： C12N15/86 , A61K45/06 , C12N15/113
摘要： The invention relates to an RNA interference (RNAi) strategy based on the use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, the invention relates to a vector that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.
-
公开(公告)号：US11427836B2
公开(公告)日：2022-08-30
申请号：US16604648
申请日：2018-04-11
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , UNIVERSITÉ D'EVRY-VAL-D'ESSONNE , GENETHON , FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA , FONDAZIONE TELETHON
IPC分类号： C12N15/113 , C12N15/86 , A61K45/06 , C12Q1/68
摘要： An RNA interference (RNAi) strategy is provided based on use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, a method is provided that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.
-
公开(公告)号：US20210130847A1
公开(公告)日：2021-05-06
申请号：US16604648
申请日：2018-04-11
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , UNIVERSITÉ D'EVRY-VAL-D'ESSONNE , GENETHON , FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA , FONDAZIONE TELETHON
IPC分类号： C12N15/86 , C12N15/113 , A61K45/06
摘要： An RNA interference (RNAi) strategy is provided based on use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, a method is provided that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.
-
公开(公告)号：US20220280655A1
公开(公告)日：2022-09-08
申请号：US17605524
申请日：2020-04-23
申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , Assistance Publique-Hôpitaux de Paris , Genethon , Sorbonne Université , Universite de Paris , Université d'Evry-Val-d'Essonne , Universite Paris XIII Paris-Nord Villetaneuse
发明人： Jessica Zucman-Rossi , Jean-Charles Nault , Tiziana La Bella , Giuseppe Ronzitti , Sandrine Imbeaud , Patrice Vidal
摘要： Adeno-associated virus (AAV) is a defective mono-stranded DNA virus, endemic in human population (35-80%). Recurrent clonal AAV2 insertions are associated with the pathogenesis of rare human hepatocellular carcinoma (HCC) developed on normal liver. The aimed of the inventors was to characterize the natural history of AAV infection in the liver. Viral DNA was thus quantified in tumor and non-tumor liver tissues of 1461 patients. Presence of episomal form and viral mRNA expression were analyzed using a DNAse/TaqMan based assay and quantitative RT-PCR. In silico analyses using viral capture data explored viral variants and new clonal insertions. AAV DNA was detected in 21% of the patients equally distributed in 2 major viral subtypes: one similar to AAV2, the other hybrid between AAV2 and AAV13 sequences. Thus the inventors provided an integrated analysis of the wild type AAV infection in the liver with the identification of viral genotypes, molecular forms, helper virus relationship and viral integrations. These findings are important to understand wild type AAV biology and particularly relevant considering the large usage of AAV vector in liver-targeted gene therapy. Thus, the present invention relates to new adeno-associated virus (AAV) variants and uses thereof for gene therapy.
-
公开(公告)号：US11541131B2
公开(公告)日：2023-01-03
申请号：US16491623
申请日：2018-03-09
申请人： GENETHON , UNIVERSITE D'EVRY VAL D'ESSONNE , INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE , SORBONNE UNIVERSITÉ
摘要： The present invention relates to vectors and compositions for the treatment of glycogen storage disease III.
-
公开(公告)号：US11421211B2
公开(公告)日：2022-08-23
申请号：US16332373
申请日：2017-09-12
申请人： GENETHON , SORBONNE UNIVERSITE , DUKE UNIVERSITY
摘要： The present invention relates to variants of acid-alpha glucosidase and uses thereof. Said variants are sequence-optimized and/or are linked to a heterogenous signal peptide.
-
公开(公告)号：US10471132B2
公开(公告)日：2019-11-12
申请号：US15303834
申请日：2015-04-27
摘要： The invention relates to a nucleic acid sequence useful in the treatment of hyperbilirubinemia, in particular in the treatment of Crigler-Najjar syndrome. More particularly, the nucleic acid sequence of the present invention is a codon-optimized UGT1A1 coding sequence.
-
8.发明公开公开(公告)号：US20230242905A1
公开(公告)日：2023-08-03
申请号：US18013339
申请日：2021-07-05
申请人： Genethon , Institut National de la Santé et de la Recherche Médicale , Universite d'Evry Val d'Essonne
发明人： Giuseppe Ronzitti , Tiziana Labella
CPC分类号： C12N15/1082 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2750/14152
摘要： The invention relates to a method of preparation of a recombinant hybrid adeno-associated virus (AAV) capsid protein with improved tropism and to the recombinant hybrid AAV capsid protein obtainable by the method. The invention relates also to the derived expression vector, modified cell, and hybrid capsid AAV vector particle packaging a gene of interest, and its use in tissue-targeted gene therapy for treating various diseases.
-
公开(公告)号：US11339406B2
公开(公告)日：2022-05-24
申请号：US16332379
申请日：2017-09-12
申请人： GENETHON , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , UNIVERSITE D'EVRY VAL D'ESSONNE
摘要： The present invention relates to variants of acid-alpha glucosidase and uses thereof.
-
-
-
-
-
-
-
-
搜索结果
9 条记录 (耗时 0.029 秒)
