公开(公告)号：US20240254462A1

公开(公告)日：2024-08-01

申请号：US18405286

申请日：2024-01-05

申请人： Omega Therapeutics, Inc.

发明人： Laura Anh Nguyen ,  Jeremiah D. Farelli ,  Adam Walter Scheidegger ,  Abigail Elizabeth Witt ,  Justin Chen ,  James Scott Sullivan

IPC分类号： C12N9/22 ,  A61K9/51 ,  A61K38/00 ,  C12N9/10 ,  C12N15/113 ,  C12N15/86

CPC分类号： C12N9/22 ,  A61K9/5123 ,  C12N9/1007 ,  C12N15/113 ,  C12N15/86 ,  A61K38/00 ,  C07K2319/81 ,  C12N2310/20 ,  C12Y305/01098

摘要： The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a secreted frizzled related protein 1 (SFRP1) gene by targeting an SFRP1 expression control region and methods of use thereof for treating an SFRP1 associated disorder, e.g., hair loss.

公开(公告)号：US20240254451A1

公开(公告)日：2024-08-01

申请号：US18436632

申请日：2024-02-08

申请人： The University of North Carolina at Chapel Hill

发明人： Eliane WAUTHIER ,  Tsunekazu OIKAWA ,  Timothy Anh-Hieu DINH ,  Praveen SETHUPATHY ,  Lola M. REID

IPC分类号： C12N5/09 ,  A01K67/0271 ,  C12Q1/6886 ,  G01N33/50 ,  G01N33/574

CPC分类号： C12N5/0693 ,  A01K67/0271 ,  C12Q1/6886 ,  G01N33/5067 ,  G01N33/57438 ,  A01K2207/12 ,  A01K2227/105 ,  C12N2503/02 ,  C12N2513/00 ,  C12Q2600/158 ,  C12Y305/01098

摘要： The present disclosure provides a model of human fibrolamellar hepatocellular carcinoma (FL-HCC) cells maintained as a transplantable tumor line in a host and a method to establish a transplantable human FL-HCC tumor line. Methods of ex vivo cultures of the FL-HCC are provided. Methods of diagnosing and treating FL-HCC tumors are also provided.

公开(公告)号：US20230190961A1

公开(公告)日：2023-06-22

申请号：US18047744

申请日：2022-10-19

申请人： University of Rochester

发明人： Steven A. GOLDMAN ,  John N. Mariani

IPC分类号： A61K48/00 ,  C12N15/86 ,  C12N9/10 ,  C12N9/80 ,  A61K38/17 ,  A61K38/50 ,  A61K38/45 ,  A61P25/28

CPC分类号： A61K48/0058 ,  C12N15/86 ,  C12N9/1007 ,  C12N9/80 ,  C12Y305/01098 ,  A61K38/1709 ,  A61K38/50 ,  A61K38/45 ,  A61K48/0066 ,  A61P25/28 ,  C12N2740/15043 ,  C12N2740/15032

摘要： The present disclosure is directed to methods of inducing rejuvenation in a population of adult glial progenitor cells, and methods of treating a subject having a myelin deficiency. The method of treating a subject having a myelin deficiency, may comprise expressing, in glial progenitor cells of the subject, one or more transcription factors selected from the group consisting of B-cell lymphoma/leukemia 11A (BCL11A), histone deacetylase 2 (HDAC2), histone-lysine N-methyltransferase EZH2 (EZH2), myc proto-oncogene protein (MYC), high mobility group protein HMGI-C (HMGA2), nuclear factor 1 B-type (NFIB), and transcriptional enhancer factor TEF-4 10 (TEAD2), wherein said expressing is effective to induce myelination in the subject, thereby treating the myelin deficiency.

公开(公告)号：US20180110755A1

公开(公告)日：2018-04-26

申请号：US15573439

申请日：2015-05-11

申请人： KANCERA AB

发明人： Johan SCHULTZ ,  Jan VÅGBERG ,  Elisabeth OLSSON ,  Katarina FÄRNEGÅRDH ,  Mattias JÖNSSON ,  Kristin HAMMER ,  Lars KRÜGER

IPC分类号： A61K31/4184 ,  C07D235/18 ,  C07D403/04 ,  C07D405/04 ,  A61P25/00 ,  A61P35/00 ,  A61P37/00

CPC分类号： A61K31/4184 ,  A61P25/00 ,  A61P35/00 ,  A61P37/00 ,  C07D235/18 ,  C07D403/04 ,  C07D405/04 ,  C12Y305/01098

摘要： A compound of formula (I) or a pharmaceutically acceptable salt thereof, and a pharmaceutical composition comprising the compound. The compound is useful in therapy, for the treatment of disorders mediated by HDAC6, such as autoimmune disorders, neurodegenerative disorders and hyperproliferative disorders, such as cancer.

公开(公告)号：US09926546B2

公开(公告)日：2018-03-27

申请号：US15249756

申请日：2016-08-29

申请人： The General Hospital Corporation

发明人： J. Keith Joung ,  Benjamin Kleinstiver ,  Vikram Pattanayak

IPC分类号： C12N9/16 ,  C12N9/22 ,  C12N15/90

CPC分类号： C12N9/22 ,  C07K2319/00 ,  C07K2319/71 ,  C12N9/16 ,  C12N15/90 ,  C12N15/902 ,  C12N15/907 ,  C12N2800/22 ,  C12Y114/11 ,  C12Y201/01043 ,  C12Y203/01048 ,  C12Y301/00 ,  C12Y305/01098

摘要： Engineered CRISPR-Cas9 nucleases with improved specificity and their use in genomic engineering, epigenomic engineering, genome targeting, and genome editing.

公开(公告)号：US09168316B2

公开(公告)日：2015-10-27

申请号：US14270358

申请日：2014-05-06

申请人： Yansong Gu ,  Hongzhe Li

发明人： Yansong Gu ,  Hongzhe Li

IPC分类号： A61K48/00 ,  C12N15/113 ,  A61K49/00 ,  A61K31/352 ,  A61K31/513 ,  A61K31/465 ,  A61K31/403 ,  A61K31/4245 ,  A61K31/166 ,  A61K31/404 ,  C12N9/80 ,  A61K31/7105 ,  A61K31/713

CPC分类号： A61K49/00 ,  A61K31/166 ,  A61K31/352 ,  A61K31/403 ,  A61K31/404 ,  A61K31/4245 ,  A61K31/465 ,  A61K31/513 ,  A61K31/7105 ,  A61K31/713 ,  C12N9/80 ,  C12Y305/01098

摘要： The present invention provides methods to reduce or inhibit weight gain by administering inhibitors of the SirT1 protein to a subject. Methods to identify such inhibitors are also disclosed.

摘要翻译： 本发明提供了通过向受试者施用SirT1蛋白质的抑制剂来减少或抑制体重增加的方法。 还公开了鉴定这些抑制剂的方法。

公开(公告)号：US20150296758A1

公开(公告)日：2015-10-22

申请号：US14648011

申请日：2013-12-03

申请人： THE JOHNS HOPKINS UNIVERSITY

发明人： Jay Bream

IPC分类号： A01K67/027 ,  C12N9/02 ,  C07K14/715 ,  C07K14/54 ,  C07K14/705 ,  C12N9/04 ,  C12N9/80

CPC分类号： A01K67/0278 ,  A01K2217/052 ,  A01K2217/056 ,  A01K2227/101 ,  A01K2227/103 ,  A01K2227/105 ,  A01K2227/108 ,  A01K2227/30 ,  A01K2227/40 ,  A01K2267/0368 ,  C07K14/54 ,  C07K14/5428 ,  C07K14/70596 ,  C07K14/7151 ,  C12N9/0006 ,  C12N9/0073 ,  C12N9/80 ,  C12N15/8509 ,  C12N2800/204 ,  C12Q1/6883 ,  C12Q1/6886 ,  C12Q2600/136 ,  C12Q2600/156 ,  C12Q2600/158 ,  C12Y101/04001 ,  C12Y305/01098

摘要： The present invention relates to the field of transgenic animals. More specifically, the present invention provides methods and composition related to humanized transgenic single polymorphism non-human animal systems. In one embodiment, a system comprises (a) a transgenic non-human animal comprising a transgene encoding a wildtype human protein, wherein the protein is biologically active in the animal; and (b) at least one transgenic non-human animal comprising a transgene encoding a variant human protein, wherein the protein is biologically active in the animal and wherein the variant comprises one or more single nucleotide polymorphisms (SNPs).

摘要翻译： 本发明涉及转基因动物领域。 更具体地，本发明提供了与人源化转基因单多态性非人动物系统相关的方法和组合。 在一个实施方案中，系统包含（a）包含编码野生型人蛋白质的转基因的转基因非人动物，其中所述蛋白质在动物中具有生物活性; 和（b）至少一种包含编码变体人蛋白质的转基因的转基因非人动物，其中所述蛋白质在动物中具有生物活性，并且其中所述变体包含一个或多个单核苷酸多态性（SNP）。

公开(公告)号：US20150110768A1

公开(公告)日：2015-04-23

申请号：US14518860

申请日：2014-10-20

申请人： Biocogent, LLC

发明人： Joseph D. Ceccoli

IPC分类号： A61K38/50 ,  A61K31/05

CPC分类号： A61K38/50 ,  A61K8/34 ,  A61K8/64 ,  A61K8/97 ,  A61K31/05 ,  A61K2800/78 ,  A61K2800/782 ,  A61Q19/08 ,  C12N9/80 ,  C12Y305/01098

摘要： The present technology relates to cosmetic and pharmaceutical compositions useful for minimizing the appearance of aging via a safe method of stimulating sirtuin activity. In particular, the present technology relates to compositions and methods that utilize a combination of a sirtuin activator with a sirtuin-offsetting agent in order to maintain a healthy population of skin cells while preventing survival and growth of abnormal cells that are irreparably damaged.

摘要翻译： 本技术涉及用于通过安全的刺激沉默调节蛋白的活性的方法使老化的外观最小化的化妆品和药物组合物。 特别地，本技术涉及利用沉默调节蛋白激活剂与沉默调节蛋白排斥剂的组合的组合物和方法，以便保持健康的皮肤细胞群，同时防止不可修复地损伤的异常细胞的存活和生长。

公开(公告)号：US20140369999A1

公开(公告)日：2014-12-18

申请号：US14306137

申请日：2014-06-16

申请人： Isis Innovation Limited

发明人： Nicholas B. La Thangue ,  Susan Fotheringham

IPC分类号： C12Q1/68 ,  G01N33/574

CPC分类号： G01N33/57496 ,  C12N15/111 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2320/10 ,  C12N2330/31 ,  C12Q1/6886 ,  C12Q2600/106 ,  C12Q2600/156 ,  C12Q2600/158 ,  C12Y305/01098 ,  G01N33/5011 ,  G01N33/574 ,  G01N33/57484 ,  G01N2333/4706 ,  G01N2333/91045 ,  G01N2333/98 ,  G01N2500/10 ,  G01N2800/52

摘要： This invention relates to a novel screening method that identifies simple molecular markers that are predictive of whether a particular disease condition is responsive to a specific treatment. Also, a method of diagnosing the susceptibility of an individual suffering from a disease to treatment with an HDAC inhibitor is provided. Also provided is a method of treating a proliferative disease or a condition which involves a change in cell differentiation or growth rate in a patient.

摘要翻译： 本发明涉及一种识别简单分子标记的新型筛选方法，其是预测特定疾病状况是否对特定治疗有反应。 另外，提供了将患有疾病的个体的敏感性诊断为用HDAC抑制剂进行治疗的方法。 还提供了治疗增殖性疾病或涉及患者细胞分化或生长速度变化的病症的方法。

公开(公告)号：US20140322136A1

公开(公告)日：2014-10-30

申请号：US14270358

申请日：2014-05-06

申请人： Yansong GU ,  Hongzhe LI

发明人： Yansong GU ,  Hongzhe LI

IPC分类号： C12N15/113 ,  A61K31/513 ,  A61K31/465 ,  A61K49/00 ,  A61K31/4245 ,  A61K31/166 ,  A61K31/404 ,  A61K38/00 ,  A61K31/352 ,  A61K31/403

CPC分类号： A61K49/00 ,  A61K31/166 ,  A61K31/352 ,  A61K31/403 ,  A61K31/404 ,  A61K31/4245 ,  A61K31/465 ,  A61K31/513 ,  A61K31/7105 ,  A61K31/713 ,  C12N9/80 ,  C12Y305/01098

摘要： The present invention provides methods to reduce or inhibit weight gain by administering inhibitors of the SirT1 protein to a subject. Methods to identify such inhibitors are also disclosed.

摘要翻译： 本发明提供了通过向受试者施用SirT1蛋白质的抑制剂来减少或抑制体重增加的方法。 还公开了鉴定这些抑制剂的方法。