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公开(公告)号：US12084658B2
公开(公告)日：2024-09-10
申请号：US17500410
申请日：2021-10-13
IPC分类号： C12N15/113 , A61P21/00 , C12N15/864 , C12Q1/68
CPC分类号： C12N15/113 , A61P21/00 , C12N15/8645 , C12Q1/68 , C12N2310/11 , C12N2320/32 , C12N2320/33 , C12N2330/51
摘要： The present invention relates to methods for shifting the splicing profile of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding U7-based small nuclear RNAs to induce DUX4 exon-skipping and the expression of shortened forms of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.
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公开(公告)号：US12048740B2
公开(公告)日：2024-07-30
申请号：US16922725
申请日：2020-07-07
发明人： Lauren O. Bakaletz
IPC分类号： A61K39/102 , A61K9/00 , A61K9/70 , A61K39/00 , A61K39/385
CPC分类号： A61K39/102 , A61K9/0014 , A61K9/7084 , A61K39/385 , A61K2039/54 , A61K2039/541 , A61K2039/545 , A61K2039/55544 , A61K2039/60 , A61K2039/6068
摘要： Provided are topical delivery systems and methods for treating and/or preventing a disease or disorder in a subject, or for eliciting an immune response in a subject including applying, to at least a portion of the subject's post-auricular region, a topical delivery system including a therapeutically effective amount of a topical pharmaceutical composition comprising at least one active agent and a pharmaceutically acceptable carrier and/or adjuvant. The topical pharmaceutical composition can include a topical immunogenic composition. The topical delivery system can further include a flexible substrate in communication with the topical immunogenic composition. The subject can be a pediatric subject.
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公开(公告)号：US12037370B2
公开(公告)日：2024-07-16
申请号：US16975602
申请日：2019-02-26
IPC分类号： C07K14/195 , A61K35/763 , A61P35/00 , C07K14/32 , C07K14/34 , C12N7/00
CPC分类号： C07K14/34 , A61K35/763 , A61P35/00 , C07K14/32 , C12N7/00 , C07K2319/55 , C07K2319/92 , C12N2710/16642
摘要： As disclosed herein, the assistance of a new generation of immunotoxins (split-immunotoxins) the range of cancers susceptible to elimination by oncolytic viruses and the specific toxicity of the latter can be expanded without compromising the selectivity of targeting. To accomplish this goal, a polypeptide chain of a potent bacterial toxin (including, but not limited to a catalytic subunit of a Diphtheria toxin, DTA) can be split into two benign parts, which can then be delivered to the cytoplasm of cancer cells via two independent cancer-specific pathways: (1) together with an oncolytic virus (encoded in its genome); (2) as a split-immunotoxin delivered via receptor-specific toxin entry pathways. The two parts are fused into a functional toxin only in the cytoplasm of dually targeted cancer cells by means of intein-catalyzed trans-splicing.
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4.发明公开公开(公告)号：US20240165056A1
公开(公告)日：2024-05-23
申请号：US18281115
申请日：2022-03-11
发明人： Jenny BARKER , Kevin BLUM
IPC分类号： A61K31/138 , A61L27/18 , A61L27/54
CPC分类号： A61K31/138 , A61L27/18 , A61L27/54 , A61L2300/204 , A61L2300/412
摘要： Disclosed are methods for treating or preventing capsular contracture or capsule formation in a subject with an implant, the method comprising administering to the subject tamoxifen or a metabolite thereof in a localized form. Also disclosed is a surgical implant coated with tamoxifen or a metabolite thereof.
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公开(公告)号：US20240164858A1
公开(公告)日：2024-05-23
申请号：US18282418
申请日：2022-03-15
发明人： David HOELZLE , Oliver F. ADUNKA , Gregory WIET
CPC分类号： A61B34/35 , A61B34/75 , A61B90/06 , A61B2034/301 , A61B2034/305 , A61B2090/064
摘要： A robotic system for cochlear implantation (CI) is described herein. The system includes a robotic tool configured to hold a cochlear implant electrode array and a controller that is operably coupled to the robotic tool. The controller includes a processor and a memory. The memory has computer-executable instructions stored thereon that, when executed by the processor, cause the processor to control the robotic tool with the degrees of freedom of a human hand.
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公开(公告)号：US20240115735A1
公开(公告)日：2024-04-11
申请号：US18273643
申请日：2022-01-26
发明人： Paul Taylor Martin
CPC分类号： A61K48/005 , A61K9/0019 , A61K45/06 , A61P1/16 , A61P3/06 , C12N9/20 , C12N15/86 , C12N2750/14143 , C12Y301/01013
摘要： The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of Lysosomal Acid Lipase Deficiency (LAL-D) disorders such as Wolman Disease and cholesterol ester storage disease (CESD). The disclosed rAAV provide a wild type lipase A (LIRA) cDNA to a subject in need which results in expression of the wild type protein.
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7.发明公开公开(公告)号：US20230255563A1
公开(公告)日：2023-08-17
申请号：US17672992
申请日：2022-02-16
IPC分类号： A61B5/00 , A61B5/055 , G01R33/341
CPC分类号： A61B5/704 , A61B5/055 , G01R33/341 , A61B2503/045
摘要： The invention relates to an insertable patient table for a Magnetic Resonance Imaging scanner, wherein the patient table has a length of up to ± 20 % of the length of the bore of the main magnet of the scanner and is adapted for pediatric and/or small patients, and wherein the patient table is adapted to be inserted into the bore of the main magnet without having a stand standing on the floor of an examination room. The invention further relates to a Magnetic Resonance Imaging scanner comprising an insertable patient table and a method for positioning a small and/or pediatric patient within the bore of a main magnet of a Magnetic Resonance Imaging scanner.
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公开(公告)号：US20230248704A1
公开(公告)日：2023-08-10
申请号：US18135460
申请日：2023-04-17
IPC分类号： A61K31/426 , A61P35/00 , A61K33/243 , A61K31/18 , A61K31/475 , A61K31/519 , A61K31/675 , A61K31/704 , A61K31/7048 , A61K38/12
CPC分类号： A61K31/426 , A61K31/18 , A61K31/475 , A61K31/519 , A61K31/675 , A61K31/704 , A61K31/7048 , A61K33/243 , A61K38/12 , A61P35/00
摘要： The present invention relates to IL-8 inhibitor compounds, preferably dual CXCR1 /CXCR2 receptor inhibitors, useful in the treatment and/or prevention of some sarcomas, preferably in the treatment and/or prevention of osteosarcoma, Ewing sarcoma, rhabdomyosarcoma or lung metastasis associated thereof.
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公开(公告)号：US20230181637A1
公开(公告)日：2023-06-15
申请号：US17910992
申请日：2021-03-11
CPC分类号： A61K35/17 , C12N5/0646 , A61K38/1774 , A61K38/2086 , A61K38/20 , A61K38/191 , A61P31/14 , C12N2501/2302 , C12N2501/2315 , C12N2501/2321 , C12N2502/30
摘要： Disclosed herein are expanded NK cells and methods of using thereof for treating, preventing, reducing, and/or inhibiting a microbial infection.
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公开(公告)号：US20230077409A1
公开(公告)日：2023-03-16
申请号：US17729751
申请日：2022-04-26
发明人： Scott Quenton Harper
IPC分类号： C12N15/113 , C07H21/02 , A61P21/00 , A61K9/00 , C12N7/00
摘要： The present invention relates to a tissue-specific promoter system for expressing microRNA (miRNA) for RNA interference-based methods of gene therapy. In these systems, the miRNA will inhibit gene expression or replace natural miRNA expression using microRNA.
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