公开(公告)号：US12004494B2

公开(公告)日：2024-06-11

申请号：US17475116

申请日：2021-09-14

申请人： Alector LLC

发明人： Arnon Rosenthal ,  Seung-Joo Lee

IPC分类号： A01K67/027 ,  A01K67/0275 ,  A01K67/0276 ,  C07K14/705 ,  C12N15/85

CPC分类号： A01K67/0276 ,  A01K67/0275 ,  C07K14/7056 ,  C07K14/70596 ,  C12N15/8509 ,  A01K2207/15 ,  A01K2217/05 ,  A01K2217/052 ,  A01K2217/07 ,  A01K2217/15 ,  A01K2227/105 ,  A01K2267/01 ,  A01K2267/03

摘要： Provided herein are transgenic non-human animals whose genomes comprise two or more human genes selected from CD33, Siglec-5, Siglec-7, Siglec-9, Siglec-11, Siglec-14, and Siglec-16, to methods of screening candidate agents that bind to and/or modulate the function and/or activity of at least one of the human genes in the transgenic non-human animals, and to methods of screening candidate agents to determine their effect on one or more activities and/or functions associated with expression of at least one of the human genes in the transgenic non-human animals. Further provided herein are methods of recapitulating a human Siglec immune system in a non-human animal, and methods of generating a non-human animal disease model comprising a human Siglec repertoire.

公开(公告)号：US12004493B2

公开(公告)日：2024-06-11

申请号：US16429978

申请日：2019-06-03

申请人： The Board of Regents of the University of Oklahoma

发明人： Hiroshi Yamada ,  Chinthalapally Rao

IPC分类号： A01K67/027 ,  A01K67/0276 ,  G01N33/50

CPC分类号： A01K67/0276 ,  G01N33/5088 ,  A01K2217/20 ,  A01K2227/105 ,  A01K2267/0312

摘要： An animal model (e.g., mouse) and method of use, and cell culture assay method, for characterizing or screening a test compound for its effect on late onset Alzheimer's disease (LOAD). The test compound may be used as a therapeutic agent for treatment of Alzheimer's disease (AD). The AD animal model may be haploinsufficient for Shugoshin 1 (Sgo1) gene, or may comprise a genetic modification enabling modulation of Sgo1 expression in the brain of the animal when exposed to an Sgo1 expression-modulating compound, such as tamoxifen. After the test compound is administered to the animal model, the presence or amount of an AD biomarker is assessed or measured.

公开(公告)号：US11993777B2

公开(公告)日：2024-05-28

申请号：US18458744

申请日：2023-08-30

申请人： Akouos, Inc.

发明人： Emmanuel John Simons ,  Ellen Reisinger ,  Sebastian Kügler ,  Hanan Al-Moyed

IPC分类号： C12N15/52 ,  A01K67/027 ,  A01K67/0276 ,  A61K38/17 ,  A61K48/00 ,  A61P27/16 ,  C07K14/47 ,  C12N5/0793 ,  C12N9/16 ,  C12N15/65 ,  C12N15/86 ,  C12N15/90

CPC分类号： C12N15/52 ,  A01K67/0276 ,  A61K38/1709 ,  A61K48/0075 ,  A61P27/16 ,  C07K14/47 ,  C12N5/062 ,  C12N9/16 ,  C12N15/65 ,  C12N15/86 ,  C12N15/902 ,  A01K2217/075 ,  A01K2227/103 ,  A01K2267/03 ,  C12N2310/20 ,  C12N2750/14143 ,  C12N2830/008 ,  C12Y301/03001

摘要： Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject.

公开(公告)号：US20240115737A1

公开(公告)日：2024-04-11

申请号：US18277023

申请日：2022-02-14

申请人： UCL Business Ltd

发明人： Joanna NG ,  Manju KURIAN ,  Simon WADDINGTON ,  John COUNSELL

IPC分类号： A61K48/00 ,  A01K67/0276 ,  A61K9/00 ,  A61P25/28 ,  C07K14/705 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A01K67/0276 ,  A61K9/0019 ,  A61K9/0085 ,  A61K48/0075 ,  A61K48/0083 ,  A61P25/28 ,  C07K14/70571 ,  C12N15/86 ,  C12N2750/14143

摘要： There is described a vector for treating Dopamine Transporter Deficiency Syndrome, the vector comprising a promoter operably linked to a human SLC6A3 gene, wherein the promoter is selected from a human synapsin 1 promoter, a CAG promoter, a CMV promoter, a CAMKII promoter, a beta-actin promoter, and a human EF1-alpha promoter. Also described are methods and uses involving the vector for treating Dopamine Transporter Deficiency Syndrome.