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1.发明公开公开(公告)号：US20240293398A1
公开(公告)日：2024-09-05
申请号：US16486223
申请日：2018-02-16
IPC分类号： A61K31/496 , A61P35/00 , C07D235/18 , C12N15/10
CPC分类号： A61K31/496 , A61P35/00 , C07D235/18 , C12N15/1065
摘要： RNA drug targets are pervasive in cells but methods to design small molecules that target them are sparse. Herein, we report a general approach to score the affinity and selectivity of RNA motif-small molecule interactions identified via selection. Named High Throughput Structure-Activity Relationships Through Sequencing (HiT-StARTS), HiT-StARTS is statistical in nature and compares input nucleic acid sequences to selected library members that bind a ligand via high throughput sequencing. The approach allowed facile definition of the fitness landscape of hundreds of thousands of RNA motif-small molecule binding partners. These results were mined against folded RNAs in the human transcriptome and identified an avid interaction between a small molecule and the Dicer nuclease-processing site in the oncogenic microRNA (miR)-18a hairpin precursor, which is a member of the miR-17-92 cluster. Application of the small molecule, Targapremir-18a, to prostate cancer cells inhibited production of miR-18a from the cluster, de-repressed serine/threonine protein kinase 4 protein (STK4), and triggered apoptosis. Profiling the cellular targets of Targapremir-18a via Chemical Cross Linking and isolation by Pull Down (Chem-CLIP), a covalent small molecule-RNA cellular profiling approach, and other studies showed specific binding of the compound to the miR-18a precursor, revealing broadly applicable factors that govern small molecule drugging of non-coding RNAs.
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公开(公告)号：US12071610B2
公开(公告)日：2024-08-27
申请号：US17140696
申请日：2021-01-04
发明人： Zhonghui Hugh Fan , Xiao Jiang , Trevor B. Tilly , John Lednicky , Chang-Yu Wu
IPC分类号： C12M1/34 , B01L3/00 , C12M1/36 , C12M3/00 , G01N33/569
CPC分类号： C12M41/46 , B01L3/527 , C12M23/44 , C12M41/48 , G01N33/56916 , G01N33/56983 , B01L2200/025 , B01L2200/026 , B01L2200/028 , B01L2400/0616
摘要： An apparatus and method are provided for performing detection of microorganisms (e.g., viruses) with high sensitivity. The apparatus and method are well suited for point-of-care (POC) testing in resource-limited regions and are capable of being operated with very little manual intervention and without the need for lab equipment. A variety of viruses can be detected with high sensitivity, including, for example, coronaviruses, Zika virus and flu viruses.
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公开(公告)号：US12065467B2
公开(公告)日：2024-08-20
申请号：US17810729
申请日：2022-07-05
IPC分类号： C12N7/00 , C07K14/005 , C12N15/86
CPC分类号： C07K14/005 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145
摘要： The present invention provides AAV capsid proteins comprising modification of one or a combination of the surface-exposed lysine, serine, threonine and/or tyrosine residues in the VP3 region. Also provided are rAAV virions comprising the AAV capsid proteins of the present invention, as well as nucleic acid molecules and rAAV vectors encoding the AAV capsid proteins of the present invention. Advantageously, the rAAV vectors and virions of the present invention have improved efficiency in transduction of a variety of cells, tissues and organs of interest, when compared to wild-type rAAV vectors and virions.
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公开(公告)号：US12060395B2
公开(公告)日：2024-08-13
申请号：US17342120
申请日：2021-06-08
CPC分类号： C07K14/4702 , A61K9/0048 , A61K38/1709 , A61K48/0075 , C07K2319/00 , C07K2319/02 , C07K2319/10 , C07K2319/50
摘要： The present invention provides methods and compositions for treating and/or preventing age related macular degeneration and other conditions involving macular degeneration, ocular neovascularization, or inflammation, including ocular inflammation. In some embodiments, the methods comprise administering an expression vector that delivers a secretable and cell penetrating Nrf2 to a subject in need thereof.
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公开(公告)号：US20240254178A1
公开(公告)日：2024-08-01
申请号：US18345226
申请日：2023-06-30
CPC分类号： C07K14/4703 , A61K9/0048 , A61K48/005 , A61K38/00 , A61K48/00 , C07K2319/02 , C07K2319/03 , C07K2319/10 , C12N15/86 , C12N2740/15041 , C12N2750/14141
摘要： The present invention provides methods and compositions for treating and/or preventing age related macular degeneration and other conditions involving macular degeneration, ocular neovascularization, or ocular inflammation. In an exemplary embodiment, a method is disclosed that involves administering an expression vector that delivers a secretable and cell penetrating CARD to a subject in need of treatment or prevention of age-related macular degeneration or another condition involving macular degeneration or ocular neovascularization.
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公开(公告)号：US20240186057A1
公开(公告)日：2024-06-06
申请号：US18528612
申请日：2023-12-04
发明人： Shuo Wang , Qinghui Huang
CPC分类号： H01F27/2823 , H01F1/0315 , H01F27/325
摘要： A transformer is provided. The transformer includes a core; a primary winding including a first wire and a second wire electrically connected in series, where the first wire and the second wire are bifilar-wound around the core; and a secondary winding including a third wire wound around the core.
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公开(公告)号：US20240181083A1
公开(公告)日：2024-06-06
申请号：US18287711
申请日：2022-04-22
发明人： Arun Srivastava , Keyun Qing , Barry John Byrne
IPC分类号： A61K48/00 , C07K14/005 , C12N15/86
CPC分类号： A61K48/0058 , A61K48/0075 , C07K14/005 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145
摘要： Provided herein are modified AAV capsid proteins, particles, nucleic acid vectors, and compositions thereof, as well as methods of their use.
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8.发明公开公开(公告)号：US20240150338A1
公开(公告)日：2024-05-09
申请号：US18261682
申请日：2022-01-14
IPC分类号： C07D417/04 , C07D277/42
CPC分类号： C07D417/04 , C07D277/42
摘要： Provided are, inter alia, compounds having a structure of Formulae (X) to (XVII), or a subordinate structure thereof, composition including the same and methods of use.
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公开(公告)号：US20240148868A1
公开(公告)日：2024-05-09
申请号：US18282503
申请日：2022-03-17
发明人： Todd Eliot Golde , Marshall S. Goodwin , Brenda Dawn Moore , Thomas B. Ladd , Yong Ran , Yona Levites , Pedro Cruz
CPC分类号： A61K39/4614 , A61K39/4633 , A61K39/4643 , A61K48/0058 , A61P25/28 , C07K14/70503 , C07K16/18 , C12N15/86 , C07K2317/622 , C07K2319/02 , C07K2319/03 , C07K2319/43 , C12N2750/14143
摘要： Provided herein are chimeric receptors, engineered cells and pharmaceutical compositions for enhancement of long-term clearance of protein aggregates in the central nervous system via phagocytosis or engulfment. Further provided herein are methods of treatment of subjects suffering from, or diagnosed with, a neurodegenerative disease by administering these receptors, modified cells, and/or pharmaceutical compositions. Administration of one or more, or a plurality of these modified cells, to a subject may provide treatment of a neurodegenerative disease such as Alzheimer's Disease (AD) or Parkinson's Disease (PD). Advantageously, the modified cells, pharmaceutical compositions, and methods of the present disclosure meet existing needs in the art by providing clearance of accumulations of protein aggregates in brain tissue in PD and AD pathologies.
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公开(公告)号：US11969468B2
公开(公告)日：2024-04-30
申请号：US17291480
申请日：2019-11-04
发明人： Roy Curtiss, III
CPC分类号： A61K39/39 , A61K35/74 , A61K39/04 , A61P37/04 , A61K2039/522 , A61K2039/523 , A61K2039/55594
摘要： Disclosed herein are unique adjuvant compositions comprising an attenuated derivative of a bacterial pathogen that undergoes lysis in vivo. In exemplary embodiments, the bacterial pathogen is a Salmonella spp. Also disclosed are methods for enhancing an immune response using the adjuvants disclosed herein.
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