公开(公告)号：US20210198636A1

公开(公告)日：2021-07-01

申请号：US16757590

申请日：2018-10-19

Applicant: Genethon ,  Institut National de la Santé et de la Recherche Médicale ,  Université d'Evry val d'Essonne

Inventor： Anne Galy ,  Youna Coquin ,  Maxime Ferrand

IPC: C12N7/04 ,  A61P11/00 ,  C12N15/113 ,  A61K48/00

Abstract: The invention relates to a pharmaceutical composition for targeting drug delivery including gene delivery to lung tissue, comprising at least a therapeutic drug or gene associated to a syncytin protein, and its use in the prevention and/or treatment of lung diseases, in particular in gene therapy of said diseases using lentiviral vector particles or lentivirus-like particles pseudotyped with syncytin protein.

公开(公告)号：US20230303411A1

公开(公告)日：2023-09-28

申请号：US18017435

申请日：2021-07-22

Applicant: Centre National de la Recherche Scientifique ,  Commissariat à l'Energie Atomique et aux Energies Alternatives ,  CY CERGY PARIS UNIVERSITE ,  Université d'Evry Val d'Essonne

Inventor： Sophie PEULON ,  Anne PENSEL

IPC: C02F1/28 ,  C25D9/08 ,  C25D17/20

CPC classification number: C02F1/288 ,  C25D9/08 ,  C25D17/20 ,  C02F2101/206

Abstract: The present invention relates to composite beads coated with H-Birnessite layered manganese oxide with a sheet nanostructure, the average thickness of the sheets being between 1 and 50 nm, and the length L of the sheets being between 0.2 μm and 3 μm, as well as the manufacturing method thereof and a cell for producing said composite beads. The present invention also relates to the use of such beads in treatments for decontaminating fluids containing toxic elements such as heavy metals or organic pollutants.

公开(公告)号：US20220184137A1

公开(公告)日：2022-06-16

申请号：US17603220

申请日：2020-04-10

Applicant: INSERM (Institut National de la Santé et de la Recherche Médicale) ,  Université d'Evry val d'Essonne ,  Genethon

Inventor： Mario AMENDOLA ,  Giulia PAVANI

IPC: A61K35/28 ,  C12N5/0789 ,  C12N15/113 ,  C07K14/805

Abstract: The present invention relates to a genetically modified hematopoietic stem cell (HSC) comprising, in at least one α-globin gene comprised in the genome thereof, at least one transgene encoding a functional β-like globin protein, the said transgene being placed under the control of the endogenous promoter of the said at least one α-globin gene.

公开(公告)号：US20210130847A1

公开(公告)日：2021-05-06

申请号：US16604648

申请日：2018-04-11

Applicant: 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

Inventor： Federico Mingozzi ,  Giuseppe Ronzitti ,  Andrea Contestabile ,  Laura Cancedda

IPC: C12N15/86 ,  C12N15/113 ,  A61K45/06

Abstract: 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.