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1. 发明申请

WO2023086939A1 COMPOSITIONS AND METHODS FOR TREATING MUCOPOLYSACCHARIDOSIS IIIA 审中-公开
公开(公告)号：WO2023086939A1
公开(公告)日：2023-05-19
申请号：PCT/US2022/079716
申请日：2022-11-11
申请人： AMICUS THERAPEUTICS, INC.
发明人： RASSOULI-TAYLOR, Leida , DO, Hung , TUSKE, Steven , BRIGNOL, Nastry
IPC分类号： C12N9/14 , A61K48/00 , A61K38/46 , C07K14/65
摘要： Provided herein are compositions and methods for treating Mucopolysaccharidosis IIIA (MPS IIIA) comprising enzyme replacement therapy and gene therapy constructs described herein.

2. 发明申请

WO2022109316A1 MULTI-TRANSGENIC PIGS WITH GROWTH HORMONE RECEPTOR KNOCKOUT FOR XENOTRANSPLANTATION 审中-公开
公开(公告)号：WO2022109316A1
公开(公告)日：2022-05-27
申请号：PCT/US2021/060165
申请日：2021-11-19
申请人： REVIVICOR, INC.
发明人： ROTHBLATT, Martine , AYARES, David , EYESTONE, Willard
IPC分类号： A01K67/027 , C07K14/475 , C07K14/65 , C07K14/705 , C07K14/74 , C07K14/71 , C12N15/85 , C12N15/90
摘要： The present disclosure is directed to transgenic animals (e.g., transgenic porcine animals) comprising multiple genetic modifications that advantageously render these animals suitable donors for xenotranspl anati on. The present disclosure extends to organs, organ fragments, tissues and cells derived from these animals and their therapeutic use. The present disclosure further extends to methods of making such animals. In certain embodiments, the transgenic animals (e.g., transgenic porcine animals) have reduced expression of the growth hormone receptor (GHR) gene or have impaired function of the GHR protein.

3. 发明申请

WO2020079489A3 TREATMENT OF NEUROPATHY WITH IGF-1-ENCODING DNA CONSTRUCTS AND HGF-ENCODING DNA CONSTRUCTS 审中-公开
公开(公告)号：WO2020079489A3
公开(公告)日：2020-04-23
申请号：PCT/IB2019/001399
申请日：2019-07-16
申请人： HELIXMITH CO., LTD.
发明人： LEE, Junghun , LEE, Nayeon , KO, Kyeong, Ryang
IPC分类号： C07K14/65 , A61K38/00 , C12N15/63 , C07K14/475
摘要： The present invention relates to a method of treating neuropathy by administering DNA constructs, encoding a human IGF-l isoform and a human HGF isoform. Further provided herein are various DNA constructs and pharmaceutical compositions including the DNA constructs that can be used for the combination therapy. The present invention provides a safe and effective way of treating neuropathic patients.

4. 发明申请

WO2019219923A1 COMPOUNDS AND METHODS FOR THE IMMOBILIZATION OF MYOSTATIN-INHIBITORS ON THE EXTRACELLULAR MATRIX BY TRANSGLUTAMINASE 审中-公开
公开(公告)号：WO2019219923A1
公开(公告)日：2019-11-21
申请号：PCT/EP2019/062830
申请日：2019-05-17
申请人： JULIUS-MAXIMILIANS-UNIVERSITÄT WÜRZBURG
发明人： MEINEL, Lorenz , LÜHMANN, Tessa , BRAUN, Alexandra
IPC分类号： C07K14/65 , A61K38/30 , C12N9/10 , A61K38/18 , C07K14/475
摘要： The present invention relates to compounds and their use in the treatment of lesions, in tissue regeneration and/or tissue engineering. The compounds act as substrates for enzymes having transglutaminase activity and are suitable for their immobilization and/or attached therapeutic or diagnostic molecules on extracellular matrix (ECM) or synthetic ECM-derived materials, in particular for the immobilization of myostatin inhibitors.

5. 发明申请

WO2019055729A1 MATERIALS AND METHODS FOR GENERATING THERAPEUTIC MESENCHYMAL STEM CELLS 审中-公开
公开(公告)号：WO2019055729A1
公开(公告)日：2019-03-21
申请号：PCT/US2018/050989
申请日：2018-09-14
申请人： UNIVERSITY OF KANSAS
发明人： NISHIMUNE, Hiroshi , BAROHN, Richard , DAWN, Buddhadeb , BADAWI, Yomna , MITCHELL, Jim , SODER, Rupal
IPC分类号： A61K35/12 , C07K14/49 , C07K14/50 , C07K14/65 , C07D401/02
摘要： Embodiments of the present disclosure relate generally to the production of therapeutic mesenchymal stem cells (MSCs). More particularly, the present disclosure relates to the use of cell culture compositions and methods for generating MSCs that secrete neurotrophic factors and synaptic organizing agents for the treatment of neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS). As such, the present disclosure addresses the need for establishing a reliable source of therapeutic stem cells useful for the treatment of neurodegenerative diseases.

6. 发明申请

WO2017047996A1 나노그를 도입한 양수 내 태아 유래 중간엽 줄기세포를 이용한 육모 촉진용 조성물의 제조방법 审中-公开
标题翻译：使用衍生自哺乳动物的胚胎的细胞干细胞促进头发生长的组合物的方法
公开(公告)号：WO2017047996A1
公开(公告)日：2017-03-23
申请号：PCT/KR2016/010177
申请日：2016-09-09
申请人： 고려대학교 산학협력단 , 주식회사 스템랩
发明人： 유승권 , 전은경 , 박정현 , 윤원진 , 손다련
IPC分类号： C12N5/073 , C07K14/475 , C07K14/49 , C07K14/50 , C07K14/65 , A61K8/98 , A61K35/50
CPC分类号： A61K8/98 , A61K35/50 , C07K14/475 , C07K14/49 , C07K14/50 , C07K14/65
摘要： 본 발명은 양수 내 태아 유래 중간엽 줄기세포의 배양액에 관한 것으로, 보다 구체적으로는 역분화 인자 나노그(Nanog)를 과발현시킨 양수 내 태아 유래 중간엽 줄기세포의 배양액을 유효성분으로 포함하는 발모 촉진 또는 탈모 방지용 조성물에 관한 것이다. 또한, 본 발명은 나노그(Nanog)가 도입된 양수 내 태아 유래 중간엽 줄기세포를 컨디션드 배지에서 배양하는 단계 및 상기 배양액을 수거하는 단계를 포함하는 상기 조성물의 제조방법에 관한 것이다. 본 발명에 따른 컨디션드 배지 조성물은 모발 성장 촉진 효과를 나타내므로, 육모 촉진용 화장료 조성물 및 약학적 조성물로써 활용이 가능하다.
摘要翻译：本发明涉及来自羊水中的胚胎的间充质干细胞的培养基，更具体地，涉及用于促进毛发生长或预防脱发的组合物，其包含作为活性成分的间充质干细胞的培养基来源于羊水中的胚胎的细胞，其中重编程因子Nanog被过表达。另外，本发明涉及一种制备该组合物的方法，包括以下步骤：在条件培养基中培养衍生自其中引入了Nanog的羊水中的胚胎的间充质干细胞; 并收集培养基。根据本发明的条件培养基组合物显示出毛发生长促进作用，因此可以用作促进毛发生长的化妆品组合物和药物组合物。

7. 发明申请

WO2016154330A1 REPORTER OF GENOMIC METHYLATION AND USES THEREOF 审中-公开
标题翻译：记录基因甲基化及其用途
公开(公告)号：WO2016154330A1
公开(公告)日：2016-09-29
申请号：PCT/US2016/023804
申请日：2016-03-23
申请人： WHITEHEAD INSTITUTE FOR BIOMEDICAL RESEARCH
发明人： STELZER, Yonatan , JAENISCH, Rudolf
IPC分类号： C12N15/09 , C12N15/11 , C07K14/65 , A61K48/00
CPC分类号： C12Q1/6897 , A61K48/00 , C07K14/65 , C12N2310/10 , C12N2310/20 , C12Q1/68 , G01N33/5023
摘要： In some aspects, described herein is a DNA methylation reporter. In some aspects, the DNA methylation reporter comprises a promoter whose activity can be affected by exogenous methylation changes without being independently regulated by the DNA methylation machinery, operably linked to a DNA sequence that encodes a reporter molecule. In some embodiments the DNA methylation reporter comprises (i) a promoter derived from a mammalian imprinted gene promoter; and (ii) a sequence that encodes a reporter molecule that is detectable in individual mammalian cells, wherein the promoter is operably linked to the sequence that encodes the reporter molecule. Also described are nucleic acids that comprise the DNA methylation reporter, cells that have the DNA methylation reporter integrated into their genome, and non-human mammals comprising cells that have the DNA methylation reporter integrated into their genome.
摘要翻译：在一些方面，本文描述的是DNA甲基化报告物。在一些方面，DNA甲基化报告基因包含启动子，该启动子的活性可受外源甲基化改变的影响，而不受DNA甲基化机制的独立调节，可操作地连接于编码报告分子的DNA序列。在一些实施方案中，DNA甲基化报告物包含（i）源自哺乳动物印记基因启动子的启动子; 和（ii）编码在各个哺乳动物细胞中可检测的报道分子的序列，其中该启动子与编码报道分子的序列有效连接。还描述了包含DNA甲基化报告物的核酸，将DNA甲基化报告基团整合到其基因组中的细胞，以及包含将DNA甲基化报告基因整合到其基因组中的细胞的非人哺乳动物。

8. 发明申请

WO2014130529A1 IN OVO ADMINISTRATION OF GROWTH FACTORS FOR IMPROVING POULTRY PERFORMANCE 审中-公开
标题翻译：在OVO管理增长因素提高管理绩效
公开(公告)号：WO2014130529A1
公开(公告)日：2014-08-28
申请号：PCT/US2014/017113
申请日：2014-02-19
申请人： ZOETIS LLC
发明人： CHARY, Parag
IPC分类号： A61K38/18 , A61K38/22 , A61K38/30 , C07K14/50 , C07K14/65
CPC分类号： C12N5/0604 , A23K20/147 , A23K50/70 , A23K50/75 , A61K9/08 , A61K38/1825 , A61K38/30 , C07K14/50 , C07K14/65
摘要： Methods for increasing breast muscle weight in poultry are disclosed herein. These methods can include delivery of compositions via in ovo administration. The methods disclosed herein also encompass the delivery of proteins such as delivery of Fibroblast Growth Factor-2 (FGF-2) or Insulin-like Growth Factor-1 (IGF-1). The methods disclosed herein are effective for increasing breast muscle weight in poultry, both in the embryo, and at slaughter age.
摘要翻译：本文公开了增加家禽胸肌重量的方法。这些方法可以包括通过卵内施用递送组合物。本文公开的方法还包括蛋白质的递送，例如递送成纤维细胞生长因子-2（FGF-2）或胰岛素样生长因子-1（IGF-1）。本文公开的方法对于在胚胎中和在屠宰时期都能增加家禽的乳房肌肉重量是有效的。

9. 发明申请

WO2014097113A2 PRODUCTION OF THERAPEUTIC PROTEINS IN GENETICALLY MODIFIED MAMMALIAN CELLS 审中-公开
标题翻译：在遗传修饰的哺乳动物细胞中生产治疗蛋白
公开(公告)号：WO2014097113A2
公开(公告)日：2014-06-26
申请号：PCT/IB2013060982
申请日：2013-12-16
申请人： NOVARTIS AG , FORNARO MARA , GLASS DAVID , JOSTOCK THOMAS , LAUX HOLGER , ROMAND SANDRINE
发明人： FORNARO MARA , GLASS DAVID , JOSTOCK THOMAS , LAUX HOLGER , ROMAND SANDRINE
IPC分类号： C07K14/65
CPC分类号： C07K14/65 , C12N15/111 , C12N15/113 , C12N15/1138 , C12N2310/14 , C12N2310/531 , C12P21/02
摘要： The invention relates to methods for the production of therapeutic proteins in mammalian cells.
摘要翻译：本发明涉及在哺乳动物细胞中产生治疗性蛋白质的方法。

10. 发明申请

WO2014085621A1 TARGETED THERAPEUTIC LYSOSOMAL ENZYME FUSION PROTEINS AND USES THEREOF 审中-公开
标题翻译：靶向治疗性LYSOSOMAL ENZYME融合蛋白及其用途
公开(公告)号：WO2014085621A1
公开(公告)日：2014-06-05
申请号：PCT/US2013/072287
申请日：2013-11-27
申请人： BIOMARIN PHARMACEUTICAL INC.
发明人： AOYAGI-SCHARBER, Mika , CHRISTIANSON, Teresa, Margaret , DVORAK-EWELL, Melita , WENDT, Daniel, J. , LONG, Shinong , LEBOWITZ, Jonathan , GOLD, Daniel, Solomon
IPC分类号： C07K14/65 , C07K7/08 , C12N1/00 , C12N15/62 , A61K38/30
CPC分类号： C07K14/65 , A61K38/00 , A61K38/47 , C07K2319/06 , C12N9/2402 , C12Y302/0105
摘要： The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases. Exemplary therapeutic fusion proteins comprise a lysosomal enzyme, a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. Also provided are compositions and methods for treating Mucopolysaccharidosis Type IIIB (Sanfilippo B Syndrome), comprising a targeted therapeutic fusion protein comprising alpha-N-acetylglucosaminidase (Naglu), a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide.
摘要翻译：本发明一般涉及可用于治疗溶酶体贮积病的治疗融合蛋白和治疗这些疾病的方法。示例性治疗性融合蛋白包含溶酶体酶，溶酶体靶向部分，例如IGF-II肽和间隔肽。还提供了用于治疗IIIB型粘多糖贮积症（Sanfilippo B综合征）的组合物和方法，其包含靶向治疗性融合蛋白，其包含α-N-乙酰氨基葡糖苷酶（Naglu），溶酶体靶向部分，例如IGF-II肽和间隔肽。

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